Materiały źródłowe

• #1 Treatment of Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3899480/

  Huntingtons disease (HD) is a dominantly inherited progressive neurological disease characterized by chorea, an involuntary brief movement that tends to flow between body regions. […] Although pharmacotherapies theoretically target these neurotransmitters, few well-conducted trials for symptomatic interventions have yielded positive results and current treatments have focused on the motor aspects of HD. Tetrabenazine is a dopamine-depleting agent that may be one of the more effective agents for reducing chorea, although it has a risk of potentially serious adverse effects. […] There are no current treatments to change the course of HD, but education and symptomatic therapies can be effective tools for clinicians to use with patients and families affected by HD. […] Although there is no established treatment to delay the onset or forestall the progression of HD, symptomatic treatment of chorea based on the neurochemical pathology known may be beneficial in some individuals, as it may have a favorable effect on motor function, quality of life, and safety.

• #2 Huntington’s disease

  https://www.nhs.uk/conditions/huntingtons-disease/treatment/

  There’s currently no cure for Huntington’s disease and no treatments that can stop the symptoms getting worse. […] The aim of treatment will be to help you manage the symptoms and stay comfortable and independent for as long as possible. […] Treatment you might be offered includes: treatment for depression, such as antidepressants and talking therapies like cognitive behavioural therapy (CBT), medicines that can help with mood and behaviour changes, medicines to help with symptoms such as twitching and jerking (chorea), physiotherapy to help with movement problems, occupational therapy to help with daily activities, speech and language therapy. […] You may also be offered genetic counselling to help you understand your diagnosis and how your family members may be affected.

• #3 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #4 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  No treatments can alter the course of Huntington’s disease. But medicines can lessen some symptoms of movement and mental health conditions. And multiple interventions can help a person adapt to changes in abilities for a certain amount of time. […] The medicines you take may change over the course of the disease, depending on your overall treatment goals. Also, medicines that treat some symptoms may result in side effects that worsen other symptoms. Treatment goals are regularly reviewed and updated. […] Medicines to treat movement disorders include: […] Medicines to control movement include tetrabenazine (Xenazine), deutetrabenazine (Austedo) and valbenazine (Ingrezza). They have been approved by the Food and Drug Administration to suppress involuntary jerking and writhing movements, known as chorea. Chorea can happen as a result of Huntington’s disease. These medicines don’t affect how the disease progresses, however. Possible side effects include drowsiness, restlessness, and the risk of worsening or triggering depression or other psychiatric conditions.

• #5 Treatment of Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3899480/

  Many agents and surgical procedures have been evaluated in HD for their efficacy in suppressing chorea, including dopamine-depleting agents, dopamine antagonists, benzodiazepines, glutamate antagonists, acetylcholinesterase inhibitors, dopamine agonists, antiseizure medications, cannabinoids, lithium, deep brain stimulation, and fetal cell transplantation. […] Despite the lack of evidence, an American Academy of Neurology Guidelines publication was recently released recommending consideration of tetrabenazine (TBZ), amantadine, or riluzole if chorea requires treatment. […] TBZ is the only US Food and Drug Administration-approved drug for HD, indicated for the treatment of chorea associated with HD. […] The efficacy of TBZ as an antichoreic drug was convincingly demonstrated in a double-blind, placebo-controlled trial conducted by the Huntington Study Group.

• #6 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  A second VMAT2 inhibitor, deutetrabenazine, was approved by the FDA in 2017. It offers similar efficacy with a potentially improved side effect profile due to its slower metabolism and more stable plasma concentrations. […] Patients who have HD and predominant features of bradykinesia and rigidity may benefit from treatment with levodopa or dopamine agonists. […] Depression in patients with HD is treatable and should be recognized promptly. Selective serotonin reuptake inhibitors (SSRIs) should be considered as first-line therapy. Other antidepressants, including bupropion, venlafaxine, nefazodone, and tricyclic antidepressants, also can be used. […] Antipsychotic medications may be necessary in patients with hallucinations, delusions, or schizophrenia-like syndromes. Newer agents, such as quetiapine, clozapine, olanzapine, and risperidone, are preferred to older agents because of the lower incidence of extrapyramidal side effects and the decreased risk for tardive syndromes.

• #7 Approved Treatments for Huntington’s Disease | Huntington’s Disease NewsEnvelope icon

  https://huntingtonsdiseasenews.com/approved-treatments-for-huntingtons-disease-2/

  There is no cure for Huntington’s disease, nor have any disease-modifying therapies been developed yet that can stop its progression. However, there are approved treatments that can help patients manage chorea — the involuntary, jerky movements that are a hallmark symptom of the rare neurodegenerative disorder. […] Austedo and Austedo XR (deutetrabenazine) are approved oral therapies used to ease chorea in adults with Huntington’s. Available in tablets, the medications are taken orally once or twice daily, depending on the formulation. Their active ingredient is similar to that of Xenazine, an older approved treatment for Huntington’s chorea, but it is designed to last longer in the body. […] Ingrezza and Ingrezza Sprinkle (valbenazine) are oral medications approved in the U.S. to reduce chorea in adults with Huntington’s. Available in capsules that can be swallowed whole or opened and sprinkled onto food, the therapies’ regimen is always one capsule per day. […] Xenazine (tetrabenazine) is an oral therapy approved to ease Huntington’s-associated chorea. Available in tablets, it is taken at an individualized dose for each patient.

• #8 Therapeutic Approaches for the Treatment of Chorea in Huntington’s Disease – touchNEUROLOGY

  https://touchneurology.com/neurodegenerative-diseases/journal-articles/therapeutic-approaches-for-the-treatment-of-chorea-in-huntingtons-disease/

  In this section, we delve into the key medications employed to manage chorea, including vesicular monoamine transporter 2 (VMAT2) inhibitors and antipsychotics. These medications exert their antichorea effects mainly through antidopaminergic mechanisms. […] Three VMAT2 inhibitors are approved by the US Food and Drug Administration (FDA) for the treatment of HD-associated chorea, including tetrabenazine (TBZ), deutetrabenazine (DTBZ) and valbenazine (VBZ). […] The pharmacokinetic and side effect profiles of TBZ led to the development of DTBZ. DTBZ is a deuterated form of TBZ with an improved pharmacokinetic profile. […] VBZ was the third medication approved by the FDA for the treatment of HD-associated chorea, gaining approval in August 2023 based on the results of the phase III, randomized, double-blind, placebo-controlled trial KINECT-HD.

• #9 Future therapies on near horizon for Huntington disease | Cleveland Clinic Journal of Medicine

  https://www.ccjm.org/page/aan-2023/huntington-therapies

  The phase II GENERATION HD-2 study will use a lower dose of tominersen with less frequent administration, and will include younger and earlier stage patients. […] Another therapeutic target is RNA interference using microRNA targeting huntingtin mRNA delivered as an intrastriatal injection. […] Gene-based therapies are exciting, but small molecules are steadily developing as well, she said. […] ANX005 is a humanized immunoglobulin G4 recombinant antibody against C1q that inhibits its function as the initiating molecule of the classical complement cascade. […] An open-label, phase II trial of ANX005 shows reduced downstream complement and neuroinflammation and improved clinical function in patients with HD. […] Valbenazine, a highly selective vesicular monoamine transporter 2 inhibitor, prevents dopamine uptake in HD. In the phase III KINECT-HD trial, valbenazine met the primary endpoint of significant improvement in chorea severity versus placebo, with improvements beginning in week 2.

• #10 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines, such as haloperidol and fluphenazine, olanzapine (Zyprexa) and aripiprazole (Abilify, Aristada) have a side effect of suppressing movements. Therefore, they may help to treat chorea. However, these medicines may worsen involuntary muscle contractions called dystonia and cause slowness of movements, resembling Parkinson’s disease. They also may cause restlessness and drowsiness. […] Other medicines that may help suppress chorea include amantadine (Gocovri), levetiracetam (Keppra, Spritam) and clonazepam (Klonopin). However, mild effectiveness and side effects may limit their use. […] Medicines to treat mental health vary depending on the conditions and symptoms. Possible treatments include: […] Antidepressants include citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac) and sertraline (Zoloft). These medicines also may have some effect on obsessive-compulsive disorder symptoms. Side effects may include nausea, diarrhea, drowsiness and low blood pressure.

• #11 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines, such as haloperidol and fluphenazine, olanzapine (Zyprexa) and aripiprazole (Abilify, Aristada) have a side effect of suppressing movements. Therefore, they may help to treat chorea. However, these medicines may worsen involuntary muscle contractions called dystonia and cause slowness of movements, resembling Parkinson’s disease. They also may cause restlessness and drowsiness. […] Other medicines that may help suppress chorea include amantadine (Gocovri), levetiracetam (Keppra, Spritam) and clonazepam (Klonopin). However, mild effectiveness and side effects may limit their use. […] Medicines to treat mental health vary depending on the conditions and symptoms. Possible treatments include: […] Antidepressants include citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac) and sertraline (Zoloft). These medicines also may have some effect on obsessive-compulsive disorder symptoms. Side effects may include nausea, diarrhea, drowsiness and low blood pressure.

• #12 Huntington’s Disease | Baylor Medicine

  https://www.bcm.edu/healthcare/specialties/neurology/parkinsons-disease-and-movement-disorders/huntingtons-disease

  More recently, a similar compound, Valbenazine (INGREZZA), also demonstrated reduction of chorea in patients with HD and was well tolerated. The medication was FDA-approved for the treatment of HD chorea in 2023. More research is needed to evaluate the long term effects of HD treatment with Valbenazine. Psychosis may improve with neuroleptics (drugs that block dopamine receptors), such as haloperidol, pimozide, fluphenazine and thioridazine. These drugs, however, can induce tardive dyskinesia and should only be used if absolutely needed to control symptoms. The atypical antipsychotics are preferred due to the lower risk of tardive dyskinesia. Clozapine, an atypical antipsychotic, may be a useful alternative to the typical neuroleptics, but the risk of agranulocytosis (a very low white cell count) complicates its use. Other atypical neuroleptics such as olanzapine (Zyprexa), quetiapine (Seroquel), and ziprasidone (Geodon) do not need close monitoring and may be easier to use but are less effective in controlling chorea. Other medications for memory loss, depression and anxiety also may be useful in some HD patients.

• #13 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines, such as haloperidol and fluphenazine, olanzapine (Zyprexa) and aripiprazole (Abilify, Aristada) have a side effect of suppressing movements. Therefore, they may help to treat chorea. However, these medicines may worsen involuntary muscle contractions called dystonia and cause slowness of movements, resembling Parkinson’s disease. They also may cause restlessness and drowsiness. […] Other medicines that may help suppress chorea include amantadine (Gocovri), levetiracetam (Keppra, Spritam) and clonazepam (Klonopin). However, mild effectiveness and side effects may limit their use. […] Medicines to treat mental health vary depending on the conditions and symptoms. Possible treatments include: […] Antidepressants include citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac) and sertraline (Zoloft). These medicines also may have some effect on obsessive-compulsive disorder symptoms. Side effects may include nausea, diarrhea, drowsiness and low blood pressure.

• #14 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines, such as haloperidol and fluphenazine, olanzapine (Zyprexa) and aripiprazole (Abilify, Aristada) have a side effect of suppressing movements. Therefore, they may help to treat chorea. However, these medicines may worsen involuntary muscle contractions called dystonia and cause slowness of movements, resembling Parkinson’s disease. They also may cause restlessness and drowsiness. […] Other medicines that may help suppress chorea include amantadine (Gocovri), levetiracetam (Keppra, Spritam) and clonazepam (Klonopin). However, mild effectiveness and side effects may limit their use. […] Medicines to treat mental health vary depending on the conditions and symptoms. Possible treatments include: […] Antidepressants include citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac) and sertraline (Zoloft). These medicines also may have some effect on obsessive-compulsive disorder symptoms. Side effects may include nausea, diarrhea, drowsiness and low blood pressure.

• #15 Approved Treatments for Huntington’s Disease | Huntington’s Disease NewsEnvelope icon

  https://huntingtonsdiseasenews.com/approved-treatments-for-huntingtons-disease/

  Benzodiazepines can also reduce chorea. They include Klonopin (clonazepam) and Valium (diazepam). […] Anti-depressants, mainly selective serotonin re-uptake inhibitors like Lexapro, Prozac, and Zoloft, are frequently used to treat Huntington’s patients’ depression. […] Doctors use anti-psychotic medications such as Seroquel (quetiapine) and Risperdal (risperidone) to treat mood disorders, including violent outbursts or delusions. […] Mood stabilizing medications can prevent the extreme highs and lows that characterize bipolar disorder. They include Carbatrol (carbamazepine), Depacon (valproate), and Lamictal (lamotrigine.) […] Physical therapy helps maintain mobility and reduce falls. […] When this happens, speech therapy can help. […] Psychotherapy can help patients and their families develop coping strategies for dealing with the disease and its progression. Occupational therapy can help patients learn to use devices that assist them in performing everyday tasks like dressing and eating.

• #16 Treatment of Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3899480/

  Other medications that are commonly considered when treating chorea include dopamine antagonists, benzodiazepines, and glutamate antagonists. […] The goal of any treatment to reduce chorea should be discussed with the patient and family. […] For patients with the akinetic form of HD (Westphal variant), antiparkinsonian medications, such as levodopa, dopamine agonists, and amantadine, may be beneficial. […] Management outside of pharmacotherapy should be considered when possible, including environmental changes. […] Until clear neuroprotective strategies are found, clinicians can address the symptoms of patients with this devastating disease.

• #17 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  A second VMAT2 inhibitor, deutetrabenazine, was approved by the FDA in 2017. It offers similar efficacy with a potentially improved side effect profile due to its slower metabolism and more stable plasma concentrations. […] Patients who have HD and predominant features of bradykinesia and rigidity may benefit from treatment with levodopa or dopamine agonists. […] Depression in patients with HD is treatable and should be recognized promptly. Selective serotonin reuptake inhibitors (SSRIs) should be considered as first-line therapy. Other antidepressants, including bupropion, venlafaxine, nefazodone, and tricyclic antidepressants, also can be used. […] Antipsychotic medications may be necessary in patients with hallucinations, delusions, or schizophrenia-like syndromes. Newer agents, such as quetiapine, clozapine, olanzapine, and risperidone, are preferred to older agents because of the lower incidence of extrapyramidal side effects and the decreased risk for tardive syndromes.

• #18 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines, such as haloperidol and fluphenazine, olanzapine (Zyprexa) and aripiprazole (Abilify, Aristada) have a side effect of suppressing movements. Therefore, they may help to treat chorea. However, these medicines may worsen involuntary muscle contractions called dystonia and cause slowness of movements, resembling Parkinson’s disease. They also may cause restlessness and drowsiness. […] Other medicines that may help suppress chorea include amantadine (Gocovri), levetiracetam (Keppra, Spritam) and clonazepam (Klonopin). However, mild effectiveness and side effects may limit their use. […] Medicines to treat mental health vary depending on the conditions and symptoms. Possible treatments include: […] Antidepressants include citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac) and sertraline (Zoloft). These medicines also may have some effect on obsessive-compulsive disorder symptoms. Side effects may include nausea, diarrhea, drowsiness and low blood pressure.

• #19 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  A second VMAT2 inhibitor, deutetrabenazine, was approved by the FDA in 2017. It offers similar efficacy with a potentially improved side effect profile due to its slower metabolism and more stable plasma concentrations. […] Patients who have HD and predominant features of bradykinesia and rigidity may benefit from treatment with levodopa or dopamine agonists. […] Depression in patients with HD is treatable and should be recognized promptly. Selective serotonin reuptake inhibitors (SSRIs) should be considered as first-line therapy. Other antidepressants, including bupropion, venlafaxine, nefazodone, and tricyclic antidepressants, also can be used. […] Antipsychotic medications may be necessary in patients with hallucinations, delusions, or schizophrenia-like syndromes. Newer agents, such as quetiapine, clozapine, olanzapine, and risperidone, are preferred to older agents because of the lower incidence of extrapyramidal side effects and the decreased risk for tardive syndromes.

• #20 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  A second VMAT2 inhibitor, deutetrabenazine, was approved by the FDA in 2017. It offers similar efficacy with a potentially improved side effect profile due to its slower metabolism and more stable plasma concentrations. […] Patients who have HD and predominant features of bradykinesia and rigidity may benefit from treatment with levodopa or dopamine agonists. […] Depression in patients with HD is treatable and should be recognized promptly. Selective serotonin reuptake inhibitors (SSRIs) should be considered as first-line therapy. Other antidepressants, including bupropion, venlafaxine, nefazodone, and tricyclic antidepressants, also can be used. […] Antipsychotic medications may be necessary in patients with hallucinations, delusions, or schizophrenia-like syndromes. Newer agents, such as quetiapine, clozapine, olanzapine, and risperidone, are preferred to older agents because of the lower incidence of extrapyramidal side effects and the decreased risk for tardive syndromes.

• #21 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #22 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  A second VMAT2 inhibitor, deutetrabenazine, was approved by the FDA in 2017. It offers similar efficacy with a potentially improved side effect profile due to its slower metabolism and more stable plasma concentrations. […] Patients who have HD and predominant features of bradykinesia and rigidity may benefit from treatment with levodopa or dopamine agonists. […] Depression in patients with HD is treatable and should be recognized promptly. Selective serotonin reuptake inhibitors (SSRIs) should be considered as first-line therapy. Other antidepressants, including bupropion, venlafaxine, nefazodone, and tricyclic antidepressants, also can be used. […] Antipsychotic medications may be necessary in patients with hallucinations, delusions, or schizophrenia-like syndromes. Newer agents, such as quetiapine, clozapine, olanzapine, and risperidone, are preferred to older agents because of the lower incidence of extrapyramidal side effects and the decreased risk for tardive syndromes.

• #23 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #24 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  Irritability may be treated with antidepressants, particularly the SSRIs; mood stabilizers, such as valproic acid or carbamazepine; and, if needed, atypical neuroleptics. […] Electroconvulsive therapy can be effective if an immediate intervention is required and in patients who do not respond to several good trials of medication. […] Other less frequent aspects of HD that may require pharmacologic treatment are mania, obsessive-compulsive disorder, anxiety, sexual disorders, myoclonus, tics, dystonia, and epilepsy.

• #25 Huntington Disease Treatment & Management: Medical Care

  https://emedicine.medscape.com/article/1150165-treatment

  Irritability may be treated with antidepressants, particularly the SSRIs; mood stabilizers, such as valproic acid or carbamazepine; and, if needed, atypical neuroleptics. […] Electroconvulsive therapy can be effective if an immediate intervention is required and in patients who do not respond to several good trials of medication. […] Other less frequent aspects of HD that may require pharmacologic treatment are mania, obsessive-compulsive disorder, anxiety, sexual disorders, myoclonus, tics, dystonia, and epilepsy.

• #26 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  A physical therapist can teach you proper and safe exercises that enhance strength, flexibility, balance and coordination. These exercises can help maintain mobility as long as possible and may reduce the risk of falls. […] An occupational therapist can assist you, your family members and caregivers on how to use assistive devices to improve function. These strategies may include: […] Handrails at home. […] Assistive devices for activities such as bathing and dressing. […] Eating and drinking utensils adapted for people with limited fine motor skills.

• #27 Physical Therapy for Huntington’s Disease | OneRehab

  https://onerehab.com/huntingtons-disease/

  During the PT sessions, the therapist can establish personalized physical therapy and exercise interventions in Huntington’s disease whose goals will be: To enhance wellness and fitness, To recommend and fit assistive devices, Maintaining respiratory capacity, To increase strength, Educating the patient and caregivers, Help with balance and gait, Create an individualized home workout program. […] For this stage, physical therapy exercises for Huntington’s disease will aim to: Keep you or your loved one mobile, Increase lost function and the quality of life, Teach strategies for promotion and relaxation, Improve the ability to walk without help, Patient and family education, Help with functioning such as transfers, Train caregivers, Introduce assistive equipment. […] Apart from physical therapy, you can benefit from occupational therapy for Huntington’s disease. It is a personalized treatment that helps a patient carry out activities of daily living. The intervention helps patients with basic skills such as walking, eating, dressing, or bathing. As a result, they enjoy improved life quality.

• #28 Physical Therapy for Huntington’s Disease – In Motion O.C.

  https://www.inmotionoc.com/ailments/non-body-part-specific/huntingtons-disease/

  The goal is to keep the patient mobile for as long as possible and reduce the risk of falls. […] Huntingtons disease physical therapy is also important for supporting proper posture. Improved posture can help reduce the severity of some involuntary and voluntary movement issues. […] Studies show that in addition to improving motor function, gait speed, and balance, Huntingtons disease physical therapy exercises have social benefits for the patient. […] Active range of motion (AROM) exercises, such as moving arm and leg joints through their full range of motion, help to prevent these contractures and should be done daily. […] Combining aerobic exercise and resistance training can help improve strength, balance, and flexibility as well as boost stamina. […] A physical therapist will introduce a variety of exercises designed to help the patient maintain and improve balance as the disease progresses. […] Physical therapy and Huntingtons disease are among our specialties at In Motion O.C.

• #29 Physical Therapy for Huntington’s Disease – In Motion O.C.

  https://www.inmotionoc.com/ailments/non-body-part-specific/huntingtons-disease/

  The goal is to keep the patient mobile for as long as possible and reduce the risk of falls. […] Huntingtons disease physical therapy is also important for supporting proper posture. Improved posture can help reduce the severity of some involuntary and voluntary movement issues. […] Studies show that in addition to improving motor function, gait speed, and balance, Huntingtons disease physical therapy exercises have social benefits for the patient. […] Active range of motion (AROM) exercises, such as moving arm and leg joints through their full range of motion, help to prevent these contractures and should be done daily. […] Combining aerobic exercise and resistance training can help improve strength, balance, and flexibility as well as boost stamina. […] A physical therapist will introduce a variety of exercises designed to help the patient maintain and improve balance as the disease progresses. […] Physical therapy and Huntingtons disease are among our specialties at In Motion O.C.

• #30 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  A physical therapist can teach you proper and safe exercises that enhance strength, flexibility, balance and coordination. These exercises can help maintain mobility as long as possible and may reduce the risk of falls. […] An occupational therapist can assist you, your family members and caregivers on how to use assistive devices to improve function. These strategies may include: […] Handrails at home. […] Assistive devices for activities such as bathing and dressing. […] Eating and drinking utensils adapted for people with limited fine motor skills.

• #31 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  A physical therapist can teach you proper and safe exercises that enhance strength, flexibility, balance and coordination. These exercises can help maintain mobility as long as possible and may reduce the risk of falls. […] An occupational therapist can assist you, your family members and caregivers on how to use assistive devices to improve function. These strategies may include: […] Handrails at home. […] Assistive devices for activities such as bathing and dressing. […] Eating and drinking utensils adapted for people with limited fine motor skills.

• #32 Physical Therapy for Huntington’s Disease | OneRehab

  https://onerehab.com/huntingtons-disease/

  Occupational therapy in the later stages of Huntington’s disease helps prevent injuries as involuntary movements increase. The therapist can recommend using padding on hazardous surfaces and using floor mats to prevent falling out of bed. They can also recommend different types of beds to help patients sleep safely.

• #33 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #34 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #35 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #36 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #37 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #38 Huntington’s disease – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/diagnosis-treatment/drc-20356122

  Antipsychotic medicines such as quetiapine (Seroquel) and olanzapine (Zyprexa) may suppress violent outbursts, agitation and other symptoms. However, these medicines may cause different movement disorders themselves. […] Mood-stabilizing medicines can help prevent the highs and lows associated with bipolar disorder. They include anti-seizure medicines such as divalproex (Depakote), carbamazepine (Tegretol, Carbatrol, Epitol, others) and lamotrigine (Lamictal). […] A psychotherapist a psychiatrist, psychologist or clinical social worker can provide talk therapy to help with behavioral symptoms. The psychotherapist can help you and your family develop coping strategies, manage expectations as the disease gets worse and help family members communicate. […] Huntington’s disease can affect the control of muscles of the mouth and throat that are essential for speech, eating and swallowing. A speech therapist can help improve your ability to speak clearly or teach you to use communication devices. A communication device might be as simple as a board covered with pictures of everyday items and activities. Speech therapists also can address trouble with eating and swallowing.

• #39 Huntington’s disease

  https://www.nhs.uk/conditions/huntingtons-disease/treatment/

  There’s currently no cure for Huntington’s disease and no treatments that can stop the symptoms getting worse. […] The aim of treatment will be to help you manage the symptoms and stay comfortable and independent for as long as possible. […] Treatment you might be offered includes: treatment for depression, such as antidepressants and talking therapies like cognitive behavioural therapy (CBT), medicines that can help with mood and behaviour changes, medicines to help with symptoms such as twitching and jerking (chorea), physiotherapy to help with movement problems, occupational therapy to help with daily activities, speech and language therapy. […] You may also be offered genetic counselling to help you understand your diagnosis and how your family members may be affected.

• #40 Huntington’s disease: diagnosis, treatment, and support | University of Iowa Health Care

  https://uihc.org/services/huntingtons-disease

  Our certified neurogenetic counselors have unique expertise in inherited neurological disorders like Huntingtons disease. We routinely coordinate genetic testing for patients and their first-degree relatives (parents, siblings, or children). […] Choosing whether to have a genetic test is a personal decision. If you’re considering it, our genetic counselors will help you understand what type of information the test provides and how it might guide your treatment decisions.

• #41 Huntington’s disease – Symptoms and causes – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/symptoms-causes/syc-20356117

  Medicines are available to help manage the symptoms of Huntington’s disease. However, treatments can’t prevent the physical, mental and behavioral decline caused by the disease. […] If an at-risk parent is considering genetic testing, it can be helpful to meet with a genetic counselor. A genetic counselor explains the potential risks of a positive test result, which may mean that the parent may develop the disease. Also, couples may need to make additional choices about whether to have children or to consider alternatives. They may decide to choose prenatal testing for the gene or in vitro fertilization with donor sperm or eggs. […] Another option for couples is in vitro fertilization and preimplantation genetic diagnosis. In this process, eggs are removed from the ovaries and fertilized with the father’s sperm in a laboratory. The embryos are tested for the presence of the Huntington gene. Only those testing negative for the Huntington gene are implanted in the mother’s uterus.

• #42 Current and Possible Future Therapeutic Options for Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9125092/

  At present, there are no approved treatments that directly target the mutated HTT gene or corresponding mHTT aggregates. Current approved HD drugs address the range of symptoms that arise as a consequence of the disease, but they do not address the disease at its origin. Each patients symptoms can be unique, hence there is no standardized treatment with medication prescribed on a case-by-case basis and an extensive range of medications used to treat each of the unique symptoms. […] The field of potential future HD therapies is advancing rapidly and holds an exciting future. Currently, attention is particularly focussed on lowering mHTT through RNA and DNA targeting therapies. Of these, antisense oligonucleotides are the most rapidly advancing potential therapies. Currently, Tominersen has one of the largest phase 3 clinical trials, and results from this trial were expected in 2022. However, the dosing in this trial has been prematurely discontinued and the results so far are being assessed.

• #43 Therapies In Pipeline – Huntington’s Disease Society of America

  https://hdsa.org/hd-research/therapies-in-pipeline/

  Tetrabenazine is used to treat the involuntary movements (chorea) of Huntington’s disease. […] Austedo is an oral medication that reduces involuntary movements in HD by lowering dopamine levels in the brain. […] Ingrezza is an oral medication that treats chorea in HD by blocking VMAT2, a protein involved in controlling dopamine release. […] Pridopidine is an oral drug that aims to protect brain cells by activating a protein called Sigma-1 Receptor (S1R), which supports cell survival and function. […] Cellavita HD is a stem cell therapy being tested in Brazil that uses cells from the soft tissue inside teeth. […] Tominersen is an investigational drug that lowers huntingtin protein by targeting its genetic message (HTT mRNA) for breakdown. […] Votoplam is an oral drug that lowers huntingtin protein by changing how the HTT gene’s message is processed, leading to its breakdown.

• #44 Current and Possible Future Therapeutic Options for Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9125092/

  Huntingtons disease (HD) is an autosomal neurodegenerative disease that is characterized by an excessive number of CAG trinucleotide repeats within the huntingtin gene (HTT). HD patients can present with a variety of symptoms including chorea, behavioural and psychiatric abnormalities and cognitive decline. Each patient has a unique combination of symptoms, and although these can be managed using a range of medications and non-drug treatments there is currently no cure for the disease. Current therapies prescribed for HD can be categorized by the symptom they treat. These categories include chorea medication, antipsychotic medication, antidepressants, mood stabilizing medication as well as non-drug therapies. Fortunately, there are also many new HD therapeutics currently undergoing clinical trials that target the disease at its origin; lowering the levels of mutant huntingtin protein (mHTT). Currently, much attention is being directed to antisense oligonucleotide (ASO) therapies, which bind to pre-RNA or mRNA and can alter protein expression via RNA degradation, blocking translation or splice modulation. Other potential therapies in clinical development include RNA interference (RNAi) therapies, RNA targeting small molecule therapies, stem cell therapies, antibody therapies, non-RNA targeting small molecule therapies and neuroinflammation targeted therapies. Potential therapies in pre-clinical development include Zinc-Finger Protein (ZFP) therapies, transcription activator-like effector nuclease (TALEN) therapies and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system (Cas) therapies. This comprehensive review aims to discuss the efficacy of current HD treatments and explore the clinical trial progress of emerging potential HD therapeutics.

• #45 CRISPR-Cas9 in Huntington’s Disease: Progress and Possibilities For Future Cure

  https://www.synthego.com/blog/crispr-huntington-research

  A myriad of approaches has been tested in clinical trials to improve treatment options for Huntington’s. Among those, antisense oligonucleotides (ASOs) are the most prominent. ASOs are short-length nucleotides (12-25 units) and block protein production from the targeted genes. […] Many scientists are currently using CRISPR-Cas9 and derivative technologies to develop novel therapies for Huntington’s disease. The two main CRISPR strategies involve either selectively knocking out the mutant HTT allele or correcting it by excising the CAG hyperexpansion, but other strategies have also recently been developed. […] The Gaj lab at the University of Illinois, Urbana, reported an effective CRISPR-based targeted attenuation of mutant HTT gene in a mouse model of the disease. […] Following on from their earlier work creating a porcine model of Huntington’s disease, researchers from Jinan University in China recently used this model to demonstrate that CRISPR-Cas9 editing can be used to correct the mutation in HTT, replacing the hyperexpansion with a normal CAG repeat. […] The hard work of scientists in labs around the world indicates that multiple Huntington’s disease therapies are possible using CRISPR technology.

• #46 Therapies In Pipeline – Huntington’s Disease Society of America

  https://hdsa.org/hd-research/therapies-in-pipeline/

  Tetrabenazine is used to treat the involuntary movements (chorea) of Huntington’s disease. […] Austedo is an oral medication that reduces involuntary movements in HD by lowering dopamine levels in the brain. […] Ingrezza is an oral medication that treats chorea in HD by blocking VMAT2, a protein involved in controlling dopamine release. […] Pridopidine is an oral drug that aims to protect brain cells by activating a protein called Sigma-1 Receptor (S1R), which supports cell survival and function. […] Cellavita HD is a stem cell therapy being tested in Brazil that uses cells from the soft tissue inside teeth. […] Tominersen is an investigational drug that lowers huntingtin protein by targeting its genetic message (HTT mRNA) for breakdown. […] Votoplam is an oral drug that lowers huntingtin protein by changing how the HTT gene’s message is processed, leading to its breakdown.

• #47 Steps to a Cure – UC Davis Huntington’s Disease Center of Excellence

  https://health.ucdavis.edu/huntingtons/steps-to-a-cure.html

  Vicki Wheelock is teaming up with stem cell scientists to develop potential cures for Huntington’s disease. […] Our plan is to conduct human clinical trials that deliver stem cells to replace damaged brain cells, reducing levels of harmful proteins that build up in the brains of Huntington’s disease patients. […] Drug therapy for Huntington’s disease is still in its infancy, said Vicki Wheelock, associate professor of neurology who directs the UC Davis Movement Disorders Clinic, a recognized center of excellence for the disease. The first drug specifically for Huntington’s disease was approved by the FDA this year, and while other clinical trials are under way, patients have no other options. […] Stem cell therapy offers the potential for a cure, not only for patients with Huntington’s disease but for those with other debilitating conditions, from Alzheimer’s disease and diabetes to osteoarthritis and spinal cord injury.

• #48 Huntington’s disease: new treatments on the horizon

  https://www.labiotech.eu/in-depth/huntingtons-disease-treatments/

  Another promising candidate for the Huntingtons community is Netherlands-based uniQures AMT-130. This one-time gene therapy is carried by an AAV5 vector and is tailored to silence the huntingtin gene to inhibit the production of mutant protein. A phase 1/2 trial reaped positive results in June. In 80% of patients who received AMT-130, disease progression slowed down. […] The candidate has been awarded orphan drug, fast track and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration (FDA), which quicken its chances of being approved. […] PTC518 arose from the biopharmas splicing platform. It is an oral small molecule that enters deep into the brain to reduce mutant Huntingtin protein levels. […] Meanwhile, Dutch biotech Prilenia Therapeutics Huntingtons candidate is en route to approval.

• #49 Therapies In Pipeline – Huntington’s Disease Society of America

  https://hdsa.org/hd-research/therapies-in-pipeline/

  Tetrabenazine is used to treat the involuntary movements (chorea) of Huntington’s disease. […] Austedo is an oral medication that reduces involuntary movements in HD by lowering dopamine levels in the brain. […] Ingrezza is an oral medication that treats chorea in HD by blocking VMAT2, a protein involved in controlling dopamine release. […] Pridopidine is an oral drug that aims to protect brain cells by activating a protein called Sigma-1 Receptor (S1R), which supports cell survival and function. […] Cellavita HD is a stem cell therapy being tested in Brazil that uses cells from the soft tissue inside teeth. […] Tominersen is an investigational drug that lowers huntingtin protein by targeting its genetic message (HTT mRNA) for breakdown. […] Votoplam is an oral drug that lowers huntingtin protein by changing how the HTT gene’s message is processed, leading to its breakdown.

• #50 What is Huntington’s Disease (HD)?

  https://prilenia.com/what-is-huntingtons-disease/

  At the present time, there is no treatment to slow progression of the disease. […] Pridopidine has now been submitted for marketing approval for the treatment of Huntington’s disease (HD) in Europe and is in late-stage development for the treatment of HD in the United States and Rest of World. […] Prilenia announced in April 2023 preliminary topline results of its Phase 3 PROOF-HD clinical study evaluating the safety and efficacy of pridopidine in individuals with Huntington’s disease (HD). […] Pridopidine was well-tolerated with no serious treatment-related adverse events, with a safety and tolerability profile similar to placebo and consistent with previous clinical studies. […] Prilenia holds orphan drug designation for pridopidine in HD in both the United States and EU. In addition, pridopidine has received Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of HD.

• #51 Therapies In Pipeline – Huntington’s Disease Society of America

  https://hdsa.org/hd-research/therapies-in-pipeline/

  Tetrabenazine is used to treat the involuntary movements (chorea) of Huntington’s disease. […] Austedo is an oral medication that reduces involuntary movements in HD by lowering dopamine levels in the brain. […] Ingrezza is an oral medication that treats chorea in HD by blocking VMAT2, a protein involved in controlling dopamine release. […] Pridopidine is an oral drug that aims to protect brain cells by activating a protein called Sigma-1 Receptor (S1R), which supports cell survival and function. […] Cellavita HD is a stem cell therapy being tested in Brazil that uses cells from the soft tissue inside teeth. […] Tominersen is an investigational drug that lowers huntingtin protein by targeting its genetic message (HTT mRNA) for breakdown. […] Votoplam is an oral drug that lowers huntingtin protein by changing how the HTT gene’s message is processed, leading to its breakdown.

• #52 Future therapies on near horizon for Huntington disease | Cleveland Clinic Journal of Medicine

  https://www.ccjm.org/page/aan-2023/huntington-therapies

  The phase II GENERATION HD-2 study will use a lower dose of tominersen with less frequent administration, and will include younger and earlier stage patients. […] Another therapeutic target is RNA interference using microRNA targeting huntingtin mRNA delivered as an intrastriatal injection. […] Gene-based therapies are exciting, but small molecules are steadily developing as well, she said. […] ANX005 is a humanized immunoglobulin G4 recombinant antibody against C1q that inhibits its function as the initiating molecule of the classical complement cascade. […] An open-label, phase II trial of ANX005 shows reduced downstream complement and neuroinflammation and improved clinical function in patients with HD. […] Valbenazine, a highly selective vesicular monoamine transporter 2 inhibitor, prevents dopamine uptake in HD. In the phase III KINECT-HD trial, valbenazine met the primary endpoint of significant improvement in chorea severity versus placebo, with improvements beginning in week 2.

• #53 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #54 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #55 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #56 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #57 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #58 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #59 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #60 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #61 Huntington’s treatment & management

  https://medically.roche.com/global/en/microsites/huntingtons-disease/diagnosis-and-symptom-management.html

  Since Huntingtons disease (HD) has such varied symptoms, it is important, when possible, for you to work closely with other healthcare professionals to create personalised care plans to address the diverse needs of individuals with HD. The goal of the multidisciplinary care team is to reduce the burden of symptoms, maximise function and optimise quality of life. […] Neurologists oversee the diagnosis, overall care coordination, neurological management and referrals to other team members during all stages of HD progression. Psychiatrists work with individuals over the course of their disease, providing overall care coordination, psychiatric management, and medications, while helping them navigate the mental healthcare system. Social workers help individuals with everything from financial and insurance planning to connecting them with support groups and HD programmes. They also provide access to community services and help manage family conflicts in the later stages of HD. Genetic counsellors work in the early stages of HD, assisting individuals with genetic testing and family counselling. Nurse specialists manage cases as well as provide telephone counselling and support for individuals with HD and their families. Dietitians work on healthy eating for HD. They make recommendations on calorie counts, food and liquid alterations, and assist with proper feeding tube supplements (if appropriate). Physical therapists develop exercise programmes and help with home preparedness and safety for the early stages of HD. As the disease progresses, they assess the need for assistive devices and the nonpharmacologic management of dystonia. Occupational therapists, if appropriate, do an assessment of the individuals workplace and their driving abilities in the early stages of HD. Once in the middle to late stages, they help with home safety, assessment of activities of daily living, and the equipment needed for seating, sleeping, feeding, hygiene, etc. Neuropsychologists oversee the management of symptoms related to personality and cognitive changes in individuals with HD. Their role ranges from counselling individuals about compensatory strategies to disability and competence assessments to family counselling in regard to cognitive changes. Psychologists offer family support and counselling on matters relating to their relationships, grief and symptom management. Speech and language therapists will conduct a pre-emptive evaluation of swallow function and provide counselling about safe swallowing in the early stages of HD. As the disease progresses, they re-evaluate dysphagia, reassess speech and communication skills, and offer individual and family counselling (as needed).

• #62 Therapies In Pipeline – Huntington’s Disease Society of America

  https://hdsa.org/hd-research/therapies-in-pipeline/

  A research “pipeline” is the process of creating, testing, and approving a new drug for use in humans. HDSA funds researchers and doctors doing Huntington’s Disease (HD) research at different stages along the pipeline, and collaborates with industry partners to bring information about clinical trials to the public. […] The research pipeline involves basic research into what goes wrong in the brain in Huntington’s disease, to identify targets for treatment. Then, new and existing drugs can be created or repurposed to address that target, and the drug development process begins. […] Once a drug is ready for testing in human patients, it must proceed through three phases of clinical trials. Phase 1 is a small trial (20-50 people with HD) testing safety. Phase 2 is a medium sized trial (50-200 people with HD) testing safety and effects of the drug on the body. Phase 3 is a large trial (200-1000 people) testing whether the drug helps with symptoms. A drug must be shown to be safe, well-tolerated, and effective before the FDA will approve it for human use.

• #63 Treatment for Huntington’s Disease | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/brain-and-nerves/huntingtons-disease/treatments.html

  Currently, no cure or treatment exists for Huntingtons disease. However, your doctor can treat your symptoms and improve your quality of life. […] Our multi-specialty team works with you to identify the best treatments for you, including: […] Medications to lessen the mental and physical effects of the disease […] Therapy for depression and anxiety that often accompany Huntingtons disease […] Exercise therapies to help movement problems […] Access to support services in your community. […] Studies of new therapies for Huntingtons disease are also in progress. Ask your doctor whether you may benefit from these treatments and if you qualify to participate.

• #64 Huntington’s Disease Specialists and Care Centers | Northwestern Medicine

  https://www.nm.org/conditions-and-care-areas/neurosciences/movement-disorders/huntingtons-disease/specialists-and-care-centers

  Northwestern Medicine Neurosciences programs provide treatment for a full range of neurological disorders, including Huntingtons disease. The clinical mission is to provide the highest quality care and treatment for patients with Huntingtons disease (HD), while advancing new therapies and uncovering the causes and cures of HD. […] Northwestern Medicine Neurosciences specialists offer comprehensive care and management of HD, including neuropsychiatric care and genetic counseling. Our goal to improve the quality of patient care in HD, advance knowledge about the disease and provide support for those dealing with the disease as well as their families. We aim to achieve this through: […] A comprehensive approach to the diagnosis and treatment of the disease, with the collaboration of specialists, including: […] Promoting clinical and basic science research to find a cure for Huntingtons disease and improve quality of life through participation in research trials. […] The HDSA Centers of Excellence provide an elite multidisciplinary approach to Huntington’s disease care and research.

• #65 Current and Possible Future Therapeutic Options for Huntington’s Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9125092/

  Patients are required to be closely monitored when taking medications and management should be patient specific. Additionally, the side effects of each drug mentioned also need to be carefully recorded and ensured they are balanced with their benefits. […] Overall, treatment of HD should not solely be the clinicians responsibility and the medications they prescribe. For optimal treatment, a range of health care professionals should be involved with an extensive network of communication between them. […] RNA therapies, particularly antisense oligonucleotides, have favourable results and are currently of the most promising future HD therapeutics. However, RNA targeting therapies come with their own challenges. It is unknown whether non-selective HTT lowering is safe, if off target effects will occur in larger populations, whether mHTT is the singular cause of HD pathology and if permanent HTT suppression is required. […] Work on future HD therapeutics will continue for numerous years to come and resulting treatments for HD patients are likely some time away. However, many of the latest developments are encouraging and a new era of HD therapeutics may be approaching.

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