Materiały źródłowe

• #1 Current State and Future Directions in the Therapy of ALS

  https://www.mdpi.com/2073-4409/12/11/1523

  Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. […] Currently, three pharmaceutical compounds with an effect on disease progression are approved and differ by country: the glutamate antagonist Riluzole (orally available in different forms: tablet, film, or liquid), the antioxidant Edaravone, and the recently introduced Sodium phenylbutyrate/Taurursodiol. By slowing down disease progression (measured by the revised ALS Functional Rating Scale, ALSFRS-R), these drugs can prolong autonomy and increase survival by a few months.

• #2 Amyotrophic Lateral Sclerosis (ALS): What It Is & Symptoms

  https://my.clevelandclinic.org/health/diseases/16729-amyotrophic-lateral-sclerosis-als

  ALS treatment includes therapies and medications to manage symptoms and slow the progression of the disease. […] Although there’s no cure, treatments are constantly improving. The right combination may slow the progression of the disease and improve your quality of life. […] No treatment can reverse ALS damage. But it may help delay the progression of symptoms and make you more comfortable. […] Your care team might recommend the following: Medications, Therapies or rehabilitation, Nutritional support, Breathing support. […] There are four medications approved by the U.S. Food and Drug Administration (FDA) to treat amyotrophic lateral sclerosis: Riluzole may reduce damage to motor neurons. It may extend survival by a few months. Edaravone can slow the decline in your muscle functioning. Sodium phenylbutyrate/taurursodiol can slow the progression of symptoms. Tofersen can decrease some damage to neurons. This may help if your provider finds a genetic change on the SOD1 gene.

• #3 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Treatments can’t reverse the damage of ALS, but they can slow the progression of symptoms. […] They also can help prevent complications and make you more comfortable and independent. […] You might need a team of health care providers and doctors trained in many areas to provide your care. The team works together to prolong your survival and improve your quality of life. […] Your team works to select the right treatments for you. You have the right to choose or refuse any of the treatments suggested. […] The Food and Drug Administration has approved two medicines for treating ALS: […] Riluzole (Rilutek, Exservan, Tiglutik). Taken by mouth, this medicine can increase life expectancy by about 25%. […] Edaravone (Radicava). This medicine may reduce the speed of decline in daily functioning.

• #4 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Treatments can’t reverse the damage of ALS, but they can slow the progression of symptoms. […] They also can help prevent complications and make you more comfortable and independent. […] You might need a team of health care providers and doctors trained in many areas to provide your care. The team works together to prolong your survival and improve your quality of life. […] Your team works to select the right treatments for you. You have the right to choose or refuse any of the treatments suggested. […] The Food and Drug Administration has approved two medicines for treating ALS: […] Riluzole (Rilutek, Exservan, Tiglutik). Taken by mouth, this medicine can increase life expectancy by about 25%. […] Edaravone (Radicava). This medicine may reduce the speed of decline in daily functioning.

• #5 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. […] Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. […] Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. […] Currently, three pharmaceutical compounds with an effect on disease progression are approved and differ by country: the glutamate antagonist Riluzole, the antioxidant Edaravone, and the recently introduced Sodium phenylbutyrate/Taurursodiol.

• #6 Current State and Future Directions in the Therapy of ALS

  https://www.mdpi.com/2073-4409/12/11/1523

  Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. […] Currently, three pharmaceutical compounds with an effect on disease progression are approved and differ by country: the glutamate antagonist Riluzole (orally available in different forms: tablet, film, or liquid), the antioxidant Edaravone, and the recently introduced Sodium phenylbutyrate/Taurursodiol. By slowing down disease progression (measured by the revised ALS Functional Rating Scale, ALSFRS-R), these drugs can prolong autonomy and increase survival by a few months.

• #7 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Treatments can’t reverse the damage of ALS, but they can slow the progression of symptoms. […] They also can help prevent complications and make you more comfortable and independent. […] You might need a team of health care providers and doctors trained in many areas to provide your care. The team works together to prolong your survival and improve your quality of life. […] Your team works to select the right treatments for you. You have the right to choose or refuse any of the treatments suggested. […] The Food and Drug Administration has approved two medicines for treating ALS: […] Riluzole (Rilutek, Exservan, Tiglutik). Taken by mouth, this medicine can increase life expectancy by about 25%. […] Edaravone (Radicava). This medicine may reduce the speed of decline in daily functioning.

• #8 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  There is no treatment to reverse damage to motor neurons or cure ALS at this time. However, some treatments may slow progression of the disease, improve quality of life, and extend survival. […] Doctors may use the following medications approved by the U.S. Food and Drug Administration (FDA) to support a treatment plan for ALS: […] Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. […] Edaravone (Radicava) is an antioxidant given either orally or intravenously and has been shown to slow functional decline in some people with ALS. […] Sodium phenylbutyrate/taurursodiol (Relyvrio) is an oral medication that was proposed to prevent nerve cell death by blocking stress signals in cells.

• #9 Amyotrophic Lateral Sclerosis

  https://practicalneurology.com/articles/2020-july-aug/amyotrophic-lateral-sclerosis-1

  There are both disease-modifying and symptom-reducing treatments available and emerging treatment possibilities. […] There is no cure; however, 2 disease-modifying treatments (DMTs) that slow disease progression are approved by the Food and Drug Administration (FDA) but unfortunately do not improve strength or function. […] A variety of potentially therapeutic medications are in clinical trials, some of which have demonstrated promising results. This article summarizes available medications to slow the disease progression and reduce symptom severity, and briefly discusses emerging treatment possibilities. […] There are 2 FDA-approved medications shown to modify the disease course of ALS: riluzole, and edaravone. […] Riluzole has been shown to play a neuroprotective role, likely through decreased glutamate transmission and inhibition of toxic excess glutamate.

• #10 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  There is no treatment to reverse damage to motor neurons or cure ALS at this time. However, some treatments may slow progression of the disease, improve quality of life, and extend survival. […] Doctors may use the following medications approved by the U.S. Food and Drug Administration (FDA) to support a treatment plan for ALS: […] Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. […] Edaravone (Radicava) is an antioxidant given either orally or intravenously and has been shown to slow functional decline in some people with ALS. […] Sodium phenylbutyrate/taurursodiol (Relyvrio) is an oral medication that was proposed to prevent nerve cell death by blocking stress signals in cells.

• #11 Current State and Future Directions in the Therapy of ALS

  https://www.mdpi.com/2073-4409/12/11/1523

  Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. […] Currently, three pharmaceutical compounds with an effect on disease progression are approved and differ by country: the glutamate antagonist Riluzole (orally available in different forms: tablet, film, or liquid), the antioxidant Edaravone, and the recently introduced Sodium phenylbutyrate/Taurursodiol. By slowing down disease progression (measured by the revised ALS Functional Rating Scale, ALSFRS-R), these drugs can prolong autonomy and increase survival by a few months.

• #12 Amyotrophic lateral sclerosis (ALS) – Diagnosis and Treatment

  https://www.uofmhealth.org/brain-neurological-conditions/als-diagnosis-treatment

  Although there is no known cure for ALS, treatment may help relieve symptoms and improve the quality of life. Treatments may include: […] We at Michigan Medicine prescribe medications that are approved for ALS including Riluzole and Edaravone. […] Riluzole: Clinical trials with ALS patients showed that Riluzole prolongs survival by several months, mainly in those with difficulty swallowing. The drug also extends the time before an individual needs ventilation support. […] Edaravone: The medicine works by relieving the effects of oxidative stress, which may be related to the death of motor neurons (nerve cells) in people with ALS. Keeping motor neurons healthy may help to preserve muscle function. […] Physicians can also prescribe medications to help reduce fatigue, ease muscle cramps, and reduce excess saliva and phlegm. Drugs also are available to help patients with pain, depression, sleep disturbances, and constipation.

• #13 Amyotrophic lateral sclerosis (ALS) – Diagnosis and Treatment

  https://www.uofmhealth.org/brain-neurological-conditions/als-diagnosis-treatment

  Although there is no known cure for ALS, treatment may help relieve symptoms and improve the quality of life. Treatments may include: […] We at Michigan Medicine prescribe medications that are approved for ALS including Riluzole and Edaravone. […] Riluzole: Clinical trials with ALS patients showed that Riluzole prolongs survival by several months, mainly in those with difficulty swallowing. The drug also extends the time before an individual needs ventilation support. […] Edaravone: The medicine works by relieving the effects of oxidative stress, which may be related to the death of motor neurons (nerve cells) in people with ALS. Keeping motor neurons healthy may help to preserve muscle function. […] Physicians can also prescribe medications to help reduce fatigue, ease muscle cramps, and reduce excess saliva and phlegm. Drugs also are available to help patients with pain, depression, sleep disturbances, and constipation.

• #14 The Management of Amyotrophic Lateral Sclerosis

  https://www.uspharmacist.com/article/the-management-of-amyotrophic-lateral-sclerosis

  Riluzole (Rilutek) is the only FDA-approved medication for the treatment of ALS. It is available as a 50-mg tablet that is taken twice daily. It is proposed that excess glutamate (an excitatory neurotransmitter) reaches toxic concentrations in the synapses, causing the neurons in the central nervous system to die. Riluzole is an antiglutamate agent that is thought to exhibit its action through direct blocking of glutamate release, noncompetitive blocking of the N-methyl-d-aspartate acid receptor, and direct action on voltage-dependent sodium channels. […] Two trials confirm that taking riluzole prolongs survival and slows disease progression. At the end of 12 and 21 months, riluzole showed a 38.6% and 19.4% reduction in mortality, respectively, when compared to placebo in the first trial. The second trial compared placebo to 50 mg, 100 mg, and 200 mg daily in divided doses. Comparing the risk-to-benefit ratio, the 100 mg daily dose (50 mg twice daily) had an acceptable safety profile and showed a 35% decrease in death or tracheostomy (a surgical airway made in the trachea) at 18 months as compared to placebo.

• #15 Medical Management – Amyotrophic Lateral Sclerosis (ALS) – Diseases | Muscular Dystrophy Association

  https://www.mda.org/disease/amyotrophic-lateral-sclerosis/medical-management

  The drug riluzole (Rilutek) has a modest effect on slowing disease progression and prolonging life. […] In 2010, Nuedexta was approved specifically for an aspect of ALS called pseudobulbar affect (PBA). […] Edaravone (Radicava) has been shown to slow the decline in clinical assessment of daily functioning in people with ALS. […] In September 2022 the FDA approved Relyvrio for the treatment of ALS. […] Most recently, on April 25, 2023, the FDA granted accelerated approval of tofersen (Qalsody) for the treatment of ALS associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). […] In addition to these disease-modifying treatments, physicians may prescribe medications to treat symptoms of ALS including drooling, anxiety and depression, constipation (the result of reduced mobility and/or weakened abdominal muscles), sleep difficulties, and pain associated with prolonged immobility. […] Talk with your MDA Care Center physician about medications and/or vitamin supplements that may be beneficial and discuss dosage levels of supplements.

• #16 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  There is no treatment to reverse damage to motor neurons or cure ALS at this time. However, some treatments may slow progression of the disease, improve quality of life, and extend survival. […] Doctors may use the following medications approved by the U.S. Food and Drug Administration (FDA) to support a treatment plan for ALS: […] Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. […] Edaravone (Radicava) is an antioxidant given either orally or intravenously and has been shown to slow functional decline in some people with ALS. […] Sodium phenylbutyrate/taurursodiol (Relyvrio) is an oral medication that was proposed to prevent nerve cell death by blocking stress signals in cells.

• #17 Amyotrophic lateral sclerosis (ALS) – Diagnosis and Treatment

  https://www.uofmhealth.org/brain-neurological-conditions/als-diagnosis-treatment

  Although there is no known cure for ALS, treatment may help relieve symptoms and improve the quality of life. Treatments may include: […] We at Michigan Medicine prescribe medications that are approved for ALS including Riluzole and Edaravone. […] Riluzole: Clinical trials with ALS patients showed that Riluzole prolongs survival by several months, mainly in those with difficulty swallowing. The drug also extends the time before an individual needs ventilation support. […] Edaravone: The medicine works by relieving the effects of oxidative stress, which may be related to the death of motor neurons (nerve cells) in people with ALS. Keeping motor neurons healthy may help to preserve muscle function. […] Physicians can also prescribe medications to help reduce fatigue, ease muscle cramps, and reduce excess saliva and phlegm. Drugs also are available to help patients with pain, depression, sleep disturbances, and constipation.

• #18 Amyotrophic Lateral Sclerosis

  https://practicalneurology.com/articles/2020-july-aug/amyotrophic-lateral-sclerosis-1

  Edaravone is thought to ameliorate ALS progression via neuroprotective elimination of free radicals. […] The many potentially disease-modifying treatments being investigated are summarized in Table 2. […] Modulation of genetic expression to promote neuronal growth and repair is an active area of investigation for potential treatments. […] The use of stem cells has emerged as a potential therapeutic technique because the primary pathogenesis of ALS is motor neuron cell death. […] Skeletal muscle activators, which activate troponin and thereby indirectly increase the calcium sensitivity of muscle sarcomeres, are being investigated for that purpose. […] Because inflammatory processes promote neuronal death in ALS, immune-modulating drugs are a prominent focus of treatment development.

• #19 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  There is no treatment to reverse damage to motor neurons or cure ALS at this time. However, some treatments may slow progression of the disease, improve quality of life, and extend survival. […] Doctors may use the following medications approved by the U.S. Food and Drug Administration (FDA) to support a treatment plan for ALS: […] Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. […] Edaravone (Radicava) is an antioxidant given either orally or intravenously and has been shown to slow functional decline in some people with ALS. […] Sodium phenylbutyrate/taurursodiol (Relyvrio) is an oral medication that was proposed to prevent nerve cell death by blocking stress signals in cells.

• #20 A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? | Translational Neurodegeneration | Full Text

  https://translationalneurodegeneration.biomedcentral.com/articles/10.1186/s40035-021-00250-5

  Edaravone is a free radical scavenger that has been approved for the treatment of ALS in a few countries (approved in Japan in 2015, South Korea in 2015, the United States in 2017, Canada in 2018, Switzerland in 2019, China in 2019, and Indonesia in 2020). Edaravone has demonstrated effects in reducing oxidative stress and delaying functional motor deterioration in a previous clinical study. However, a phase III confirmatory trial (MCI 18616) has failed to demonstrate the efficacy of edaravone in the prolongation of survival and respiratory function improvement. […] ALS patients suffer from a wide range of debilitating symptoms, including fatigue, cramps, spasticity, dysphagia, respiratory insufficiency, sleep disorders, pain, psychosocial morbidity, etc. Supportive care plays an important role in providing symptom management and improving quality of life. The symptoms of ALS could be relieved by pharmacological and non-pharmacological interventions. Thus, multidisciplinary ALS care alleviating the symptoms can enhance the quality of life and prolong the survival of patients.

• #21 Medical Management – Amyotrophic Lateral Sclerosis (ALS) – Diseases | Muscular Dystrophy Association

  https://www.mda.org/disease/amyotrophic-lateral-sclerosis/medical-management

  The drug riluzole (Rilutek) has a modest effect on slowing disease progression and prolonging life. […] In 2010, Nuedexta was approved specifically for an aspect of ALS called pseudobulbar affect (PBA). […] Edaravone (Radicava) has been shown to slow the decline in clinical assessment of daily functioning in people with ALS. […] In September 2022 the FDA approved Relyvrio for the treatment of ALS. […] Most recently, on April 25, 2023, the FDA granted accelerated approval of tofersen (Qalsody) for the treatment of ALS associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). […] In addition to these disease-modifying treatments, physicians may prescribe medications to treat symptoms of ALS including drooling, anxiety and depression, constipation (the result of reduced mobility and/or weakened abdominal muscles), sleep difficulties, and pain associated with prolonged immobility. […] Talk with your MDA Care Center physician about medications and/or vitamin supplements that may be beneficial and discuss dosage levels of supplements.

• #22 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Nutritional therapy can help maintain body weight and respiratory failure with the resulting hypercapnia can be treated by noninvasive or invasive ventilation, a method that can prolong survival. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. […] We further discuss the ongoing clinical trials with a particular focus on small molecules, but also clinical trials focusing on gene-specific therapy (antisense oligonucleotides and viral vectors delivering RNA interference and CRISPR/Cas9) as well as monoclonal antibody and stem-cell therapy. […] Sodium phenylbutyrate and Taurursodiol have been released as a fixed-dose combination. […] The combination of both substances slowed down disease progression (measured by the ALSFRS-R) by about 25% compared to the placebo group, and initiation of PB-TURSO treatment at baseline resulted in prolonged median survival of about 6.5 months.

• #23 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Nutritional therapy can help maintain body weight and respiratory failure with the resulting hypercapnia can be treated by noninvasive or invasive ventilation, a method that can prolong survival. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. […] We further discuss the ongoing clinical trials with a particular focus on small molecules, but also clinical trials focusing on gene-specific therapy (antisense oligonucleotides and viral vectors delivering RNA interference and CRISPR/Cas9) as well as monoclonal antibody and stem-cell therapy. […] Sodium phenylbutyrate and Taurursodiol have been released as a fixed-dose combination. […] The combination of both substances slowed down disease progression (measured by the ALSFRS-R) by about 25% compared to the placebo group, and initiation of PB-TURSO treatment at baseline resulted in prolonged median survival of about 6.5 months.

• #24 Current State and Future Directions in the Therapy of ALS

  https://www.mdpi.com/2073-4409/12/11/1523

  The combination of both substances slowed down disease progression (measured by the ALSFRS-R) by about 25% compared to the placebo group, and initiation of PB-TURSO treatment at baseline resulted in prolonged median survival of about 6.5 months. […] Supportive and palliative care is highly important to patients but rarely focused on in research. Nonmotor symptoms involving neuropsychiatric, autonomic, gastrointestinal, and vascular systems affect between 5% and 80% of people with ALS. Thus, symptomatic treatment forms an important part of therapy in ALS patients and must be adapted to the clinical course. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. In this field, more studies are necessary in order to objectify the relevant effects. The vast number of substances currently tested, as well as the rising number of clinical trials within the last years, give high hopes for future therapeutic options to treat, or possibly cure, ALS.

• #25 Medical Management – Amyotrophic Lateral Sclerosis (ALS) – Diseases | Muscular Dystrophy Association

  https://www.mda.org/disease/amyotrophic-lateral-sclerosis/medical-management

  The drug riluzole (Rilutek) has a modest effect on slowing disease progression and prolonging life. […] In 2010, Nuedexta was approved specifically for an aspect of ALS called pseudobulbar affect (PBA). […] Edaravone (Radicava) has been shown to slow the decline in clinical assessment of daily functioning in people with ALS. […] In September 2022 the FDA approved Relyvrio for the treatment of ALS. […] Most recently, on April 25, 2023, the FDA granted accelerated approval of tofersen (Qalsody) for the treatment of ALS associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). […] In addition to these disease-modifying treatments, physicians may prescribe medications to treat symptoms of ALS including drooling, anxiety and depression, constipation (the result of reduced mobility and/or weakened abdominal muscles), sleep difficulties, and pain associated with prolonged immobility. […] Talk with your MDA Care Center physician about medications and/or vitamin supplements that may be beneficial and discuss dosage levels of supplements.

• #26 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  Tofersen (Qalsody) is given through a spinal injection to people with ALS who have been determined to have a mutation in the SOD1 gene. […] A treatment plan for ALS usually includes rehabilitation, which should be tailored to the persons individual needs and may include physical, occupational, and speech therapy. […] Physical therapy can help the person with ALS maintain function, including lowering their risk of falls and joint pain and maximizing their independence at different stages of the disease. […] Speech therapists can help people with ALS learn strategies to speak louder and more clearly and help maintain the ability to communicate. […] Nutritionists and registered dieticians can help plan small, nutritious meals throughout the day and identify foods to avoid. […] Noninvasive ventilation (NIV) is a type of breathing support that is usually delivered through a mask over the nose and/or mouth. […] There are several techniques to increase forceful coughing, including mechanical cough assistive devices.

• #27 Amyotrophic Lateral Sclerosis (ALS) Treatment | ALS News TodayEnvelope icon

  https://alsnewstoday.com/als-treatment/

  Edaravone was approved by the FDA in 2017, marking the first new approval for an ALS therapy in more than two decades. […] Relyvrio (sodium phenylbutyrate and taurursodiol) is a fixed-dose combination of two compounds that work together to reduce cellular stress pathways and promote nerve cell survival. […] Qalsody (tofersen) is an RNA-based therapy designed to reduce the amount of toxic SOD1, a protein that builds up and forms toxic clumps in some ALS patients. […] There are no stem cell therapies currently approved to treat amyotrophic lateral sclerosis (ALS), although several stem cell-based treatments are in clinical development. […] Nonetheless, several approved therapies have been proven to slow the progression of ALS, allowing patients to maintain a higher level of functionality for longer. […] There also are medications that can be used to help manage specific symptoms of ALS, such as spasticity or excess saliva production.

• #28 ALS – Wikipedia

  https://en.wikipedia.org/wiki/ALS

  Tofersen is an antisense oligonucleotide that was approved for medical use in the United States in April 2023, for the treatment of SOD1-associated ALS. In a study of 108 patients with SOD1-associated ALS there was a non-significant trend towards a slowing of progression, as well as a significant reduction in neurofilament light chain, a putative ALS biomarker thought to indicate neuronal damage.

• #29 A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? | Translational Neurodegeneration | Full Text

  https://translationalneurodegeneration.biomedcentral.com/articles/10.1186/s40035-021-00250-5

  Edaravone is a free radical scavenger that has been approved for the treatment of ALS in a few countries (approved in Japan in 2015, South Korea in 2015, the United States in 2017, Canada in 2018, Switzerland in 2019, China in 2019, and Indonesia in 2020). Edaravone has demonstrated effects in reducing oxidative stress and delaying functional motor deterioration in a previous clinical study. However, a phase III confirmatory trial (MCI 18616) has failed to demonstrate the efficacy of edaravone in the prolongation of survival and respiratory function improvement. […] ALS patients suffer from a wide range of debilitating symptoms, including fatigue, cramps, spasticity, dysphagia, respiratory insufficiency, sleep disorders, pain, psychosocial morbidity, etc. Supportive care plays an important role in providing symptom management and improving quality of life. The symptoms of ALS could be relieved by pharmacological and non-pharmacological interventions. Thus, multidisciplinary ALS care alleviating the symptoms can enhance the quality of life and prolong the survival of patients.

• #30 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #31 ALS | Conditions | UCSF Health

  https://www.ucsfhealth.org/conditions/als

  Proper medication and physical therapy, however, can help control symptoms and allow patients to lead more independent lives. […] Although there is no known cure for ALS, the drug riluzole has been approved for treatment and may slow progression of the disease. It is expensive, however, and appears modestly effective. Generally, treatment is designed to help control symptoms. […] Medications prescribed include: […] Drugs such as baclofen or diazepam may help control spasticity. […] Gabapentin may be prescribed to help control pain. […] Trihexyphenidyl or amitriptyline may help patients swallow saliva. […] Because choking is common as ALS progresses, patients may need a tube called a percutaneous endoscopic gastrostomy (PEG) tube or feeding tube to be placed into the stomach for feeding.

• #32 Amyotrophic Lateral Sclerosis

  https://practicalneurology.com/articles/2020-july-aug/amyotrophic-lateral-sclerosis-1

  Tauroursodeoxycholic acid (TUDCA) inhibits programmed cell death and significantly reduced ALSFRS-R score decline in a phase 2 clinical trial. […] Treatments for symptoms of ALS include a combination of dextromethorphan and quinidine for pseudobulbar affect. […] First-line treatments for sialorrhea are anticholinergic drugs such as scopolamine, glycopyrrolate, atropine, hyoscyamine, and amitriptyline. […] Baclofen and tizanidine are commonly used to treat spasticity, although there is limited evidence supporting either in alleviating ALS-specific spasticity symptoms. […] Pain and discomfort from muscle cramping is a common concern for those with ALS. […] Fatigue is a ubiquitous symptom of ALS, present in as many as 90% of people with ALS.

• #33 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  Tofersen (Qalsody) is given through a spinal injection to people with ALS who have been determined to have a mutation in the SOD1 gene. […] A treatment plan for ALS usually includes rehabilitation, which should be tailored to the persons individual needs and may include physical, occupational, and speech therapy. […] Physical therapy can help the person with ALS maintain function, including lowering their risk of falls and joint pain and maximizing their independence at different stages of the disease. […] Speech therapists can help people with ALS learn strategies to speak louder and more clearly and help maintain the ability to communicate. […] Nutritionists and registered dieticians can help plan small, nutritious meals throughout the day and identify foods to avoid. […] Noninvasive ventilation (NIV) is a type of breathing support that is usually delivered through a mask over the nose and/or mouth. […] There are several techniques to increase forceful coughing, including mechanical cough assistive devices.

• #34 Amyotrophic Lateral Sclerosis (ALS): What It Is & Symptoms

  https://my.clevelandclinic.org/health/diseases/16729-amyotrophic-lateral-sclerosis-als

  Your provider might recommend different types of therapy or rehabilitation, including: Physical therapy helps you stay independent and safe through gentle exercises that strengthen muscles and support overall health. Occupational therapy teaches strategies for daily tasks and using assistive devices like wheelchairs or braces without tiring yourself out. Speech therapy supports safe swallowing and communication, helping you speak as long as possible and conserve energy with nonverbal options. […] A dietitian can help you follow a meal plan that avoids hard-to-swallow foods and provides the right balance of calories, fiber and fluids. Nutritional counseling supports healthy eating, and a nutritionist can suggest alternatives as swallowing becomes more challenging. […] As ALS progresses, breathing may become difficult. Noninvasive ventilation (NIV), delivered through a mask over your nose and mouth, can make breathing more comfortable. You may start using it at night and eventually need it full time.

• #35 Are There Any Treatments for ALS?

  https://www.webmd.com/brain/understanding-als-treatment

  Dextromethorphan HBr and Quinidine Sulfate (Nuedexta): This medication is used to treat a certain mental/mood disorder (pseudobulbar affect) that may accompany ALS. […] Tofersen (Qalsody): This medication is given by injection into the spine. […] Riluzole (Exservan, Rilutek): taken orally, it helps reduce damage to your motor nerves by reducing the amount of glutamate in your system. […] Most treatments for ALS involve managing the symptoms of the disease as it worsens. […] Physical therapy and exercise: These keep your muscles strong and working as long as possible. […] Hot tub and whirlpool baths: These can ease your muscle spasms or cramps. […] Dietary counseling: This becomes really important when swallowing becomes a problem. […] Speech therapy: Specialists can help you learn ways to make your speech more clear when you talk or other methods of communicating, such as writing with pen and paper or an alphabet board.

• #36 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #37 Amyotrophic Lateral Sclerosis (ALS): What It Is & Symptoms

  https://my.clevelandclinic.org/health/diseases/16729-amyotrophic-lateral-sclerosis-als

  Your provider might recommend different types of therapy or rehabilitation, including: Physical therapy helps you stay independent and safe through gentle exercises that strengthen muscles and support overall health. Occupational therapy teaches strategies for daily tasks and using assistive devices like wheelchairs or braces without tiring yourself out. Speech therapy supports safe swallowing and communication, helping you speak as long as possible and conserve energy with nonverbal options. […] A dietitian can help you follow a meal plan that avoids hard-to-swallow foods and provides the right balance of calories, fiber and fluids. Nutritional counseling supports healthy eating, and a nutritionist can suggest alternatives as swallowing becomes more challenging. […] As ALS progresses, breathing may become difficult. Noninvasive ventilation (NIV), delivered through a mask over your nose and mouth, can make breathing more comfortable. You may start using it at night and eventually need it full time.

• #38 Are There Any Treatments for ALS?

  https://www.webmd.com/brain/understanding-als-treatment

  Occupational therapy: This can help you find ways to dress, bathe, and groom. […] There are also a variety of tools and mechanical devices that can help if you have ALS: […] Splints, reach extenders, and grab-bars: They can help you get around as the disease progresses. […] Canes, walkers, and wheelchairs: They can help you stay mobile even as your ability to walk fades. […] Computerized voice synthesizers: These are available when you lose the ability to speak. […] Respirator: It may be needed in the late stage of the disease to help you breathe. […] Feeding tube: As swallowing becomes harder, you might also need a doctor to insert a feeding tube into your stomach.

• #39 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  Tofersen (Qalsody) is given through a spinal injection to people with ALS who have been determined to have a mutation in the SOD1 gene. […] A treatment plan for ALS usually includes rehabilitation, which should be tailored to the persons individual needs and may include physical, occupational, and speech therapy. […] Physical therapy can help the person with ALS maintain function, including lowering their risk of falls and joint pain and maximizing their independence at different stages of the disease. […] Speech therapists can help people with ALS learn strategies to speak louder and more clearly and help maintain the ability to communicate. […] Nutritionists and registered dieticians can help plan small, nutritious meals throughout the day and identify foods to avoid. […] Noninvasive ventilation (NIV) is a type of breathing support that is usually delivered through a mask over the nose and/or mouth. […] There are several techniques to increase forceful coughing, including mechanical cough assistive devices.

• #40 Amyotrophic lateral sclerosis (ALS) – Diagnosis and Treatment

  https://www.uofmhealth.org/brain-neurological-conditions/als-diagnosis-treatment

  Physical and occupational therapy: Physical therapy and special equipment can enhance an individuals independence and safety throughout the course of ALS. Gentle, low-impact aerobic exercise such as walking, swimming, and stationary bicycling can strengthen unaffected muscles, improve cardiovascular health, and help patients fight fatigue and depression. […] Speech therapy: As ALS progresses, speech therapists can help people develop ways for responding to yes-or-no questions with their eyes or by other nonverbal means and can recommend aids such as speech synthesizers and computer-based communication systems. These methods and devices help people communicate when they can no longer speak or produce vocal sounds. […] Breathing devices: When the muscles that assist in breathing weaken, use of nocturnal ventilatory assistance may be used to aid breathing during sleep. These devices may be used full-time as the disease reaches more advanced stage.

• #41 Are There Any Treatments for ALS?

  https://www.webmd.com/brain/understanding-als-treatment

  Dextromethorphan HBr and Quinidine Sulfate (Nuedexta): This medication is used to treat a certain mental/mood disorder (pseudobulbar affect) that may accompany ALS. […] Tofersen (Qalsody): This medication is given by injection into the spine. […] Riluzole (Exservan, Rilutek): taken orally, it helps reduce damage to your motor nerves by reducing the amount of glutamate in your system. […] Most treatments for ALS involve managing the symptoms of the disease as it worsens. […] Physical therapy and exercise: These keep your muscles strong and working as long as possible. […] Hot tub and whirlpool baths: These can ease your muscle spasms or cramps. […] Dietary counseling: This becomes really important when swallowing becomes a problem. […] Speech therapy: Specialists can help you learn ways to make your speech more clear when you talk or other methods of communicating, such as writing with pen and paper or an alphabet board.

• #42 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #43 Amyotrophic Lateral Sclerosis (ALS) | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als

  Tofersen (Qalsody) is given through a spinal injection to people with ALS who have been determined to have a mutation in the SOD1 gene. […] A treatment plan for ALS usually includes rehabilitation, which should be tailored to the persons individual needs and may include physical, occupational, and speech therapy. […] Physical therapy can help the person with ALS maintain function, including lowering their risk of falls and joint pain and maximizing their independence at different stages of the disease. […] Speech therapists can help people with ALS learn strategies to speak louder and more clearly and help maintain the ability to communicate. […] Nutritionists and registered dieticians can help plan small, nutritious meals throughout the day and identify foods to avoid. […] Noninvasive ventilation (NIV) is a type of breathing support that is usually delivered through a mask over the nose and/or mouth. […] There are several techniques to increase forceful coughing, including mechanical cough assistive devices.

• #44 Treatments for ALS: What Are the Options?

  https://www.healthline.com/health/treatments-for-als

  Several other medications may also be recommended to manage troublesome symptoms like painful muscle spasms. […] Since breathing eventually becomes a central issue in ALS, options for respiratory support are discussed with a healthcare professional early in your treatment plan. This is because weakness of your breathing muscles can ultimately lead to respiratory failure. […] There are a few different options to support breathing with ALS: positive pressure ventilation, tracheostomy (a tube is surgically placed into your trachea), ventilator. […] Edaravone is a relatively new medication thats sometimes recommended to taken with riluzole. Its an antioxidant that helps to protect your nerves from degeneration. Its not been shown to improve survival time, but it may improve your function according to a 2018 review.

• #45 Patient Care and Treatment in Amyotrophic Lateral Sclerosis | Ikeda | Journal of Neurology Research

  https://www.neurores.org/index.php/neurores/article/view/176/183

  Patient care and therapy of ALS has considerably amended over the past two decades. Recently, three effective treatments have been recommended for prolonging survival in ALS patients: multidisciplinary team care, mechanical ventilation and gastrostomy tubes. […] This review introduces current treatment in ALS patients at the following four divisions: symptomatic treatment; ventilation; nutrition; and disease modifying medication. […] The AAN practice advisory recommended riluzole at 100 mg/day to extend survival in ALS patients clinically probable or definite El Escorial ALS who had symptoms less than 5 years, FVC 60% and non-tracheostomy. […] The AAN Practice Parameter of ALS care recommends that NIV should be considered to treat respiratory insufficiency in ALS patients for lengthening survival and slowing FVC decline rate. […] Most guidelines address that supplementary enteral feeding should be considered when body weight loss (BMI) 10% of the pre-diagnostic or baseline weight. […] Therefore, further studies investigating high nutrition diets and iron chelating are warranted for the treatment of ALS.

• #46 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #47 Amyotrophic Lateral Sclerosis (ALS): What It Is & Symptoms

  https://my.clevelandclinic.org/health/diseases/16729-amyotrophic-lateral-sclerosis-als

  Your provider might recommend different types of therapy or rehabilitation, including: Physical therapy helps you stay independent and safe through gentle exercises that strengthen muscles and support overall health. Occupational therapy teaches strategies for daily tasks and using assistive devices like wheelchairs or braces without tiring yourself out. Speech therapy supports safe swallowing and communication, helping you speak as long as possible and conserve energy with nonverbal options. […] A dietitian can help you follow a meal plan that avoids hard-to-swallow foods and provides the right balance of calories, fiber and fluids. Nutritional counseling supports healthy eating, and a nutritionist can suggest alternatives as swallowing becomes more challenging. […] As ALS progresses, breathing may become difficult. Noninvasive ventilation (NIV), delivered through a mask over your nose and mouth, can make breathing more comfortable. You may start using it at night and eventually need it full time.

• #48 Amyotrophic lateral sclerosis (ALS) – Symptoms and causes – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/symptoms-causes/syc-20354022

  ALS causes both groups of motor neurons to gradually deteriorate and then die. […] There is no cure for this fatal disease. […] A feeding tube can reduce these risks and ensure proper hydration and nutrition. […] Researchers continue to study possible causes of ALS. Most theories center on a complex interaction between genes and factors in the environment. […] The most common cause of death for people with ALS is breathing failure. […] Some people with advanced ALS choose to have a tracheostomy. This is a surgically created hole at the front of the neck leading to the windpipe. A ventilator may work better on a tracheostomy than on a mask. […] Over time, ALS leads to weakness of the muscles used to breathe. People with ALS might need a device such as a mask ventilator to help them breathe at night.

• #49 Patient Care and Treatment in Amyotrophic Lateral Sclerosis | Ikeda | Journal of Neurology Research

  https://www.neurores.org/index.php/neurores/article/view/176/183

  Patient care and therapy of ALS has considerably amended over the past two decades. Recently, three effective treatments have been recommended for prolonging survival in ALS patients: multidisciplinary team care, mechanical ventilation and gastrostomy tubes. […] This review introduces current treatment in ALS patients at the following four divisions: symptomatic treatment; ventilation; nutrition; and disease modifying medication. […] The AAN practice advisory recommended riluzole at 100 mg/day to extend survival in ALS patients clinically probable or definite El Escorial ALS who had symptoms less than 5 years, FVC 60% and non-tracheostomy. […] The AAN Practice Parameter of ALS care recommends that NIV should be considered to treat respiratory insufficiency in ALS patients for lengthening survival and slowing FVC decline rate. […] Most guidelines address that supplementary enteral feeding should be considered when body weight loss (BMI) 10% of the pre-diagnostic or baseline weight. […] Therefore, further studies investigating high nutrition diets and iron chelating are warranted for the treatment of ALS.

• #50 Amyotrophic lateral sclerosis (ALS) – Diagnosis and Treatment

  https://www.uofmhealth.org/brain-neurological-conditions/als-diagnosis-treatment

  Feeding tube: When individuals can no longer get enough nourishment from eating, doctors may advise inserting a feeding tube into the stomach. The use of a feeding tube also reduces the risk of choking and pneumonia that can result from inhaling liquids into the lungs. […] PEG tubes help people to live well, remain well nourished, and well hydrated. They greatly reduce the work of eating, and weight loss can be stopped or reversed. Some people can return to their usual body weight after insertion. All our muscles and organs require good energy from good nutrition, including our breathing muscles. PEG tubes can help to improve quality and length of life. Good nutrition and hydration gives your body what it needs to fight the disease, repair damage and to try to grow new nerves. It is the best fight anyone has against ALS.

• #51 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #52 Amyotrophic Lateral Sclerosis (ALS): Causes, Symptoms and Treatment

  https://www.apollohospitals.com/diseases-and-conditions/amyotrophic-lateral-sclerosis-als-types-causes-symptoms-treatment

  The doctor may also provide medications that give relief from other symptoms, such as muscle cramps and spasms, constipation, fatigue, excessive saliva and phlegm, pain, depression, sleep issues, and uncontrolled emotions, such as laughing or crying. […] As the disease progresses, the patient may find it challenging to breathe independently. […] A physical therapist may help a patient practice low-impact exercises to maintain cardiovascular fitness, muscle strength, and a range of motions for a prolonged period. […] Occupational therapy helps the patient be independent regardless of hand and arm weakness. […] The therapist teaches the patient with ALS adaptive techniques to ensure the speech is more understandable. […] The doctors will work closely with the patient and their families to list foods that can be easily swallowed and meet the necessary dietary requirements. […] A team of social workers and psychologists for financial issues and emotional support.

• #53 Amyotrophic lateral sclerosis (ALS) – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/diagnosis-treatment/drc-20354027

  Your health care provider also might prescribe treatments for relief of other symptoms, including: […] When ALS affects your ability to breathe, speak and move, therapies and other forms of support can help. […] Most people with ALS eventually have more trouble breathing as muscles weaken. […] A physical therapist can address pain, walking, mobility, bracing and equipment needs that help you stay independent. […] An occupational therapist can help you find ways to remain independent despite hand and arm weakness. […] A speech therapist can teach you adaptive techniques to make your speech more understandable. […] Your team typically works with you and your family members to ensure you are eating foods that are easier to swallow and meet your nutritional needs. […] Your team might include a social worker to help with financial issues, insurance, and getting equipment and paying for devices you need. […] Based on the current understanding of ALS, researchers are conducting clinical studies on promising medicines and treatments.

• #54 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Nutritional therapy can help maintain body weight and respiratory failure with the resulting hypercapnia can be treated by noninvasive or invasive ventilation, a method that can prolong survival. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. […] We further discuss the ongoing clinical trials with a particular focus on small molecules, but also clinical trials focusing on gene-specific therapy (antisense oligonucleotides and viral vectors delivering RNA interference and CRISPR/Cas9) as well as monoclonal antibody and stem-cell therapy. […] Sodium phenylbutyrate and Taurursodiol have been released as a fixed-dose combination. […] The combination of both substances slowed down disease progression (measured by the ALSFRS-R) by about 25% compared to the placebo group, and initiation of PB-TURSO treatment at baseline resulted in prolonged median survival of about 6.5 months.

• #55 A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? | Translational Neurodegeneration | Full Text

  https://translationalneurodegeneration.biomedcentral.com/articles/10.1186/s40035-021-00250-5

  Autophagy, a catabolic and recycling process that eliminates dysfunctional organelles and abnormal protein aggregates in the cell, is essential to neuronal homeostasis. Impaired autophagy represents a critical pathomechanism in ALS, and autophagic regulation is therefore emerging as a potential therapeutic strategy for ALS. […] Accumulating evidence has supported the idea that transplantation of stem cells may become a promising alternative therapy for ALS. Transplanted stem cells could secrete growth factors such as glial cell-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor (BDNF), vascular endothelial growth factor (VEGF), and insulin-like growth factor-1 to provide neurotrophic support and slow the degeneration of MNs. […] Gene therapies are used to replace or correct a defective gene involved in disease pathogenesis. The latest strategies developed to suppress the expression of mutated genes include anti-sense oligonucleotides (ASOs), RNA interference (RNAi) and CRISPR-Cas9 genome editing system.

• #56 A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? | Translational Neurodegeneration | Full Text

  https://translationalneurodegeneration.biomedcentral.com/articles/10.1186/s40035-021-00250-5

  Autophagy, a catabolic and recycling process that eliminates dysfunctional organelles and abnormal protein aggregates in the cell, is essential to neuronal homeostasis. Impaired autophagy represents a critical pathomechanism in ALS, and autophagic regulation is therefore emerging as a potential therapeutic strategy for ALS. […] Accumulating evidence has supported the idea that transplantation of stem cells may become a promising alternative therapy for ALS. Transplanted stem cells could secrete growth factors such as glial cell-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor (BDNF), vascular endothelial growth factor (VEGF), and insulin-like growth factor-1 to provide neurotrophic support and slow the degeneration of MNs. […] Gene therapies are used to replace or correct a defective gene involved in disease pathogenesis. The latest strategies developed to suppress the expression of mutated genes include anti-sense oligonucleotides (ASOs), RNA interference (RNAi) and CRISPR-Cas9 genome editing system.

• #57 Amyotrophic lateral sclerosis stem cell therapy: What to know

  https://www.medicalnewstoday.com/articles/amyotrophic-lateral-sclerosis-stem-cell-therapy

  The Food and Drug Administration (FDA) has not approved stem cell therapy for amyotrophic lateral sclerosis (ALS). However, people could participate in clinical trials involving this ALS treatment. […] Stem cell therapy for ALS may offer different benefits. […] According to the ALS Association, medical professionals may administer stem cells, which can deliver growth factors or protective molecules to nerve cells. This can help improve the health of the nerve cells and prevent them from dying. […] Stem cell therapy may also use stem cells to replace damaged nerve cells. However, this may be challenging because the stem cells will need to mimic the appropriate connections between the surrounding neurons and muscles. […] The FDA has not currently approved stem cell therapy for ALS. This means that a person can only make use of this treatment if they take part in a clinical trial.

• #58 Advancements in Pharmacological Interventions and Novel Therapeutic Approaches for Amyotrophic Lateral Sclerosis

  https://www.mdpi.com/2227-9059/12/10/2200

  Edaravone, approved in 2017 by the FDA, was developed by Mitsubishi Tanabe Pharma in Jersey City, NJ, USA. […] Given the complex symptomatology of ALS, it is essential to explore non-pharmacological interventions to maximize patients’ functional capacity. […] Non-invasive mechanical ventilation (NIV) remains the most widely used therapeutic modality, as it improves quality of life, increases survival, and preserves forced vital capacity in ALS patients, as corroborated by clinical trials. […] Nutritional interventions have also shown promise. A randomized trial demonstrated the benefits of high-calorie nutritional intake in ALS patients. […] Another therapy involves the use of mesenchymal stem cells to help slow the progression of the disease. […] The need for developing novel therapies and strategies to treat ALS more effectively and efficiently is evident. Among the drugs discussed in this systematic review, only riluzole and edaravone have demonstrated significant positive outcomes, delaying disease progression by several months.

• #59 A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? | Translational Neurodegeneration | Full Text

  https://translationalneurodegeneration.biomedcentral.com/articles/10.1186/s40035-021-00250-5

  Autophagy, a catabolic and recycling process that eliminates dysfunctional organelles and abnormal protein aggregates in the cell, is essential to neuronal homeostasis. Impaired autophagy represents a critical pathomechanism in ALS, and autophagic regulation is therefore emerging as a potential therapeutic strategy for ALS. […] Accumulating evidence has supported the idea that transplantation of stem cells may become a promising alternative therapy for ALS. Transplanted stem cells could secrete growth factors such as glial cell-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor (BDNF), vascular endothelial growth factor (VEGF), and insulin-like growth factor-1 to provide neurotrophic support and slow the degeneration of MNs. […] Gene therapies are used to replace or correct a defective gene involved in disease pathogenesis. The latest strategies developed to suppress the expression of mutated genes include anti-sense oligonucleotides (ASOs), RNA interference (RNAi) and CRISPR-Cas9 genome editing system.

• #60 Amyotrophic Lateral Sclerosis

  https://practicalneurology.com/articles/2020-july-aug/amyotrophic-lateral-sclerosis-1

  Edaravone is thought to ameliorate ALS progression via neuroprotective elimination of free radicals. […] The many potentially disease-modifying treatments being investigated are summarized in Table 2. […] Modulation of genetic expression to promote neuronal growth and repair is an active area of investigation for potential treatments. […] The use of stem cells has emerged as a potential therapeutic technique because the primary pathogenesis of ALS is motor neuron cell death. […] Skeletal muscle activators, which activate troponin and thereby indirectly increase the calcium sensitivity of muscle sarcomeres, are being investigated for that purpose. […] Because inflammatory processes promote neuronal death in ALS, immune-modulating drugs are a prominent focus of treatment development.

• #61 Treatments for ALS: What Are the Options?

  https://www.healthline.com/health/treatments-for-als

  Relyvrio is another medication that was recently approved by the Food and Drug Administration (FDA) for ALS. It was shown to slow ALS progression in a small clinical trial performed in 2020. […] Several other potential therapies are currently being studied for their use in ALS, although none so far have shown to be beneficial. Therapies are nonetheless being aggressively pursued. […] According to 2022 research, some therapies currently undergoing research include: stem cells, tofersen, tadnersen, jacifusen. […] Working with a team of healthcare professionals can help improve your quality of life. Its essential to get treatment as early as possible and to stick with it. As research continues, more treatments are expected to become available.

• #62 Medical Management – Amyotrophic Lateral Sclerosis (ALS) – Diseases | Muscular Dystrophy Association

  https://www.mda.org/disease/amyotrophic-lateral-sclerosis/medical-management

  Medical interventions and technology have vastly improved the quality of life for people with ALS by assisting with breathing, nutrition, mobility, and communication. […] Managing the symptoms is the mainstay treatment for ALS; multidisciplinary care may improve quality of life. […] In October 2009, the American Academy of Neurology (AAN) released revised physician guidelines for medical management of ALS. […] The physician may recommend noninvasive ventilation to compensate for weakened muscles by assisting the movement of air in and out of the lungs. […] It is recommended that patients with ALS receive pneumococcal vaccination and annual seasonal influenza vaccination as they may have compromised ability to handle respiratory secretions and an increased risk of developing chronic pulmonary conditions.

• #63 Patient Care and Treatment in Amyotrophic Lateral Sclerosis | Ikeda | Journal of Neurology Research

  https://www.neurores.org/index.php/neurores/article/view/176/183

  Patient care and therapy of ALS has considerably amended over the past two decades. Recently, three effective treatments have been recommended for prolonging survival in ALS patients: multidisciplinary team care, mechanical ventilation and gastrostomy tubes. […] This review introduces current treatment in ALS patients at the following four divisions: symptomatic treatment; ventilation; nutrition; and disease modifying medication. […] The AAN practice advisory recommended riluzole at 100 mg/day to extend survival in ALS patients clinically probable or definite El Escorial ALS who had symptoms less than 5 years, FVC 60% and non-tracheostomy. […] The AAN Practice Parameter of ALS care recommends that NIV should be considered to treat respiratory insufficiency in ALS patients for lengthening survival and slowing FVC decline rate. […] Most guidelines address that supplementary enteral feeding should be considered when body weight loss (BMI) 10% of the pre-diagnostic or baseline weight. […] Therefore, further studies investigating high nutrition diets and iron chelating are warranted for the treatment of ALS.

• #64 Advancements in Pharmacological Interventions and Novel Therapeutic Approaches for Amyotrophic Lateral Sclerosis

  https://www.mdpi.com/2227-9059/12/10/2200

  (1) Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease in which the patient suffers from an affection of both upper and lower motor neurons at the spinal and brainstem level, causing a progressive paralysis that leads to the patient’s demise. […] Despite the fact that a cure for ALS is currently unknown, there exists a variety of pharmacological and non-pharmacological therapies that can help reduce the progression of the disease over a certain period of time and alleviate symptoms. (2) We aim to analyze these pharmacological and non-pharmacological therapies through a systematic review. A comprehensive, multidisciplinary approach to treatment is necessary. (3) Drugs such as riluzole, edaravone, and sodium phenylbutyrate, among others, have been investigated. Additionally, it is important to stay updated on research on new drugs, such as masitinib, from which very good results have been obtained. (4) Therapies aimed at psychological support, speech and language, and physical therapy for the patient are also available, which increase the quality of life of the patients.

• #65 Amyotrophic Lateral Sclerosis Treatment & Management: Approach Considerations, Inpatient and Outpatient Care, Pharmacologic Treatment

  https://emedicine.medscape.com/article/1170097-treatment

  Treatment of amyotrophic lateral sclerosis (ALS) may be divided broadly into the following: Patient education, Mechanism-specific treatment, Adaptive or supportive treatment. […] Patient education can be enhanced by referral to multidisciplinary clinics staffed by specialists with special interest in ALS, by educational materials prepared for patients and families by national organizations in the United States and other countries or by individual experts, and by patient participation in local support groups. […] Most of the care of patients with ALS may be delivered in the outpatient setting. Often, guidance can be provided by a neurologist, physiatrist, or palliative care physician with special interest in the disease. […] Multidisciplinary clinics can provide „one-stop shopping”, allowing patients to receive all assessments and recommendations in the course of a single visit; most of these clinics provide a combination of on-site and off-site services.

• #66 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. […] Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. […] Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. […] Currently, three pharmaceutical compounds with an effect on disease progression are approved and differ by country: the glutamate antagonist Riluzole, the antioxidant Edaravone, and the recently introduced Sodium phenylbutyrate/Taurursodiol.

• #67 Current State and Future Directions in the Therapy of ALS

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10252394/

  Supportive and palliative care is highly important to patients but rarely focused on in research. […] Access to palliative care can improve the quality of life of patients and primary caregivers. […] There are constant efforts for technological improvement in order to help preserve patients independence; however, there are few studies objectifying the benefits and possible disadvantages of robotics. […] The field of robotics is also highly interesting regarding the topic of bulbar symptoms. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. […] The vast number of substances currently tested, as well as the rising number of clinical trials within the last years, give high hopes for future therapeutic options to treat, or possibly cure, ALS.

• #68 Current State and Future Directions in the Therapy of ALS

  https://www.mdpi.com/2073-4409/12/11/1523

  The combination of both substances slowed down disease progression (measured by the ALSFRS-R) by about 25% compared to the placebo group, and initiation of PB-TURSO treatment at baseline resulted in prolonged median survival of about 6.5 months. […] Supportive and palliative care is highly important to patients but rarely focused on in research. Nonmotor symptoms involving neuropsychiatric, autonomic, gastrointestinal, and vascular systems affect between 5% and 80% of people with ALS. Thus, symptomatic treatment forms an important part of therapy in ALS patients and must be adapted to the clinical course. […] Until a satisfactory causative treatment is found, supportive therapy remains of the utmost importance during disease accompaniment. In this field, more studies are necessary in order to objectify the relevant effects. The vast number of substances currently tested, as well as the rising number of clinical trials within the last years, give high hopes for future therapeutic options to treat, or possibly cure, ALS.

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