Materiały źródłowe

• #1 Myasthenia Gravis Treatment & Management: Approach Considerations, Pharmacologic Therapy, Management of neonatal myasthenia gravis

  https://emedicine.medscape.com/article/1171206-treatment

  Even though no rigorously tested treatment trials have been reported and no clear consensus exists on treatment strategies, myasthenia gravis (MG) is one of the most treatable neurologic disorders. Several factors (eg, severity, distribution, rapidity of disease progression) should be considered before therapy is initiated or changed. Treatment regimens are individualized depending on the severity of the myasthenia (MGFA class), patient age, serology status, thymic pathology, concurrent medical issues, patient and physician preference and physician experience. […] In October 2013, the Myasthenia Gravis Foundation of America appointed a Task Force to develop treatment guidance for MG. Definitions were developed for goals of treatment, minimal manifestation status (MMS), remission, ocular MG, impending crisis, crisis, and refractory MG. Guidance statements were developed for symptomatic and immunosuppressive treatments, IV immunoglobulin and plasma exchange, management of impending and manifest myasthenic crisis, thymectomy, juvenile MG, MG associated with antibodies to muscle-specific tyrosine kinase, and MG in pregnancy.

• #2 What are Treatment Options for Myasthenia Gravis (MG)?

  https://myastheniagravis.org/about-mg/treatments/

  Myasthenia gravis (MG) can be treated with drugs, surgery and other therapies alone or in combination. Whats right for you depends on the severity of your disease, which muscles are affected, your age, and the presence of other medical problems. […] MG treatment also includes self-care: getting plenty of sleep, resting your eyes, pacing your activity, eating healthy foods, exercising, and managing your stress. […] There are two types of medications used to treat MG. One group anticholinesterases temporarily relieves the symptoms of MG. Another group immunosuppressants attacks the disease at its source. […] Doctors may prescribe one or a combination of these medications. It often takes time to determine the best medication and dosage for an individual patient. […] This is typically the first type of medication prescribed because it is has the fewest long-term side effects. It also is the most rapid-acting medication available.

• #2 What are Treatment Options for Myasthenia Gravis (MG)?

  https://myastheniagravis.org/about-mg/treatments/

  The most commonly prescribed medication of this type is pyridostigmine bromide (Mestinon). […] Immunosuppressants help prevent your body from producing the harmful antibodies that cause MG weakness in the first place. […] The use of these medications must be monitored carefully by a physician because they may cause major side effects. […] Prednisone significantly relieves MG symptoms for a large majority of those with myasthenia. […] Doctors recommend thymectomies for individuals with thymomas, and for most patients under age 60 with moderate to severe generalized myasthenia. […] According to the National Institute of Neurological Disorders and Stroke, surgery reduces MG symptoms and may cure some people, possibly by re-balancing the immune system. […] Immune globulin therapy can be used to treat rapidly worsening MG.

• #2 What are Treatment Options for Myasthenia Gravis (MG)?

  https://myastheniagravis.org/about-mg/treatments/

  Plasmapheresis, or plasma exchange, is a procedure that removes antibodies both bad and good from the blood. […] Plasmapheresis is used for patients who have severe MG symptoms, or who need to improve strength before surgery. […] MG can be fought with traditional medicine. But how else can you take a measure of control over your illness? […] Talk to your doctor to see if a diet change will help ease medication side effects like fluid retention, bone loss or anemia. […] Exercise. It boosts your mood as well as the health of your heart, brain and cardiovascular system. […] Consult with your doctor and consider options such as acupuncture, biofeedback, meditation, or massage therapy.

• #3 Myasthenia gravis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myasthenia-gravis/diagnosis-treatment/drc-20352040

  Various treatments, alone or together, can help with symptoms of myasthenia gravis. Your treatment will depend on your age, how severe your disease is and how fast it’s progressing. […] Medicines such as pyridostigmine (Mestinon, Regonal) improve communication between nerves and muscles. These medicines aren’t a cure, but they can improve muscle contraction and muscle strength in some people. […] Corticosteroids such as prednisone (Rayos) block the immune system, making it less able to produce antibodies. […] Your provider also might prescribe other medicines that change your immune system. These medicines could include azathioprine (Azasan, Imuran), mycophenolate mofetil (Cellcept), cyclosporine (Sandimmune, Gengraf, others), methotrexate (Trexall) or tacrolimus (Astagraf XL, Prograf, others).

• #3 Myasthenia gravis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myasthenia-gravis/diagnosis-treatment/drc-20352040

  The following therapies are usually used for a short time to treat symptoms that suddenly get worse or before surgery or other therapies. […] This procedure uses a filtering process that’s like dialysis. Your blood is put through a machine that removes the antibodies that block transmission of signals from your nerve endings to your muscles. […] This therapy provides your body with typical antibodies, which alters your immune system response. […] Rituximab (Rituxan) and eculizumab (Soliris) are medicines given by vein for myasthenia gravis. […] If you have a tumor, called a thymoma, you’ll need surgery to remove the thymus gland, called thymectomy. […] Even if you don’t have a tumor in the thymus gland, removing the gland might improve your symptoms. However, the benefits of this surgery can take years to develop. […] These procedures might cause less blood loss, less pain, lower mortality rates and shorter hospital stays compared with open surgery.

• #4

  https://www.nhs.uk/conditions/myasthenia-gravis/treatment/

  Treatment can help keep the symptoms of myasthenia gravis under control so that you’re able to live a largely normal life. […] But some people need ongoing treatment, and occasionally emergency treatment in hospital may be necessary if the condition suddenly gets worse. […] The first medicine used for myasthenia gravis is usually a tablet called pyridostigmine, which helps electrical signals travel between the nerves and muscles. […] If pyridostigmine does not help or only provides short-term relief, your doctor may suggest taking steroid tablets such as prednisolone. […] If steroids are not controlling your symptoms, or you need to take a high dose of steroids, or steroids cause significant side effects, your doctor may suggest taking a different medicine that reduces the activity of your immune system, such as azathioprine or mycophenolate.

• #4

  https://www.nhs.uk/conditions/myasthenia-gravis/treatment/

  Surgery to remove the thymus gland, known as a thymectomy, may sometimes be recommended if you have myasthenia gravis. […] Some people with myasthenia gravis have periods where their symptoms get suddenly worse for example, they may experience severe breathing or swallowing problems. […] Treatment may include: oxygen through a face mask, using a breathing machine (ventilator), intravenous immunoglobulin therapy a treatment made from donated blood, which improves muscle strength by temporarily changing how your immune system works, plasmapheresis where your blood is circulated through a machine that filters out the harmful antibodies that are attacking the communication system between the nerves and muscles.

• #5 Treating myasthenia gravis beyond the eye clinic | Eye

  https://www.nature.com/articles/s41433-024-03133-x

  Cholinesterase inhibitors such as pyridostigmine (usual dose 3060mg three to four times a day, titrating up if needed up to 360mg/day in divided doses) can produce rapid relief of symptoms in mild MG and are usually prescribed as the initial therapy. […] Majority of patients require suppression of autoantibody production with immunosuppressive treatment which remains the cornerstone of MG therapy. Oral corticosteroids are initially used to induce remission while long-term maintenance is achieved with either low dose oral corticosteroids or non-steroidal immunosuppressants. […] Azathioprine, mycophenolate mofetil, methotrexate, ciclosporin and tacrolimus are the most frequently used non-steroidal agents. […] Rapidly acting, but short-lasting agents such as therapeutic plasma exchange (PLEX) or intravenous immunoglobulin (IVIg) are used in patients with impending respiratory crisis and severe MG.

• #5 Treating myasthenia gravis beyond the eye clinic | Eye

  https://www.nature.com/articles/s41433-024-03133-x

  In all MG patients who have a thymoma or those who are AChR antibody positive, therapeutic thymectomy is recommended. […] Rituximab is a chimeric mouse/human antiCD20 mAb which rapidly depletes the mature and memory B cells in the peripheral blood while largely sparing the pre-B cells and plasma cells located in the bone marrow and secondary lymphoid organs. […] The traditional treatment plan in MG used to be to start low and titrate up slowly. This can cause significant morbidity from mainly steroid-related side effects affecting the quality of life. […] Inhibition of complement pathway and neonatal Fc receptor (FcRn) are two of the most successful mechanisms identified among them. […] Eculizumab is a humanised monoclonal antibody (mAb) directed against the C5 component of the complement pathway.

• #5 Treating myasthenia gravis beyond the eye clinic | Eye

  https://www.nature.com/articles/s41433-024-03133-x

  Ravulizumab is a long-acting C5 complement inhibitor with a mechanism of action similar to eculizumab, which only needs fewer infusions. […] Zilucoplan is a small macrocyclic peptide molecule which prevents the terminal activation of the complement cascade by binding to C5 complement component. […] Autologous hematopoietic stem cell transplantation (HSCT) is being increasingly used for eliminating disease activity in various autoimmune neurological conditions including MG.

• #6 What are the treatment options for myasthenia gravis if first-line agents fail? | Cleveland Clinic Journal of Medicine

  https://www.ccjm.org/content/90/2/81

  If the patient with myasthenia gravis (MG) has been taking adequate doses of a first-line medication, typically pyridostigmine, for a sufficient duration but without significant efficacy, or has experienced substantial adverse effects, it may be time to consider immunosuppressive therapy. […] For these patients, nonsteroidal immunosuppressive therapy should be considered early instead of continuing high-dose corticosteroids for a longer duration. […] When pyridostigmine doses exceed 120 mg every 3 hours, or a total daily dose of 960 mg, adverse effects including risk of cholinergic crisis tend to outweigh benefits. However, if a patient needs more than 240 mg of pyridostigmine per day, it is usually beneficial to move on to immunotherapy. […] Patients with limited symptoms such as mild ptosis and facial weakness who respond well to pyridostigmine may not need immunosuppressive agents or thymectomy.

• #7 Myasthenia Gravis: What You Need To Know | Patient Care

  https://weillcornell.org/news/myasthenia-gravis-what-you-need-to-know

  The treatment for myasthenia gravis involves two main components. First, a medication called pyridostigmine blocks the enzymes from breaking down the neurotransmitter. Although this treatment is a great symptomatic quick fix, it doesn’t change the underlying cause of the disease. […] Thats where the second component of treatment comes in: immunosuppression. A number of long-term treatments can suppress the immune system and the symptoms of myasthenia gravis, allowing the muscles to function more normally. Steroids are tried-and-true options that can help most patients feel better within a few weeks. But while effective, long-term use of steroids can bring on undesired side effects. […] To avoid those side effects, doctors may choose to use a number of steroid-sparing medications. Some are oral, including medications that have been around for quite some timeazathioprine and mycophenolate. The oral medications are typically taken daily to treat the disease and suppress symptoms.

• #8 Myasthenia Gravis Treatment & Management: Approach Considerations, Pharmacologic Therapy, Management of neonatal myasthenia gravis

  https://emedicine.medscape.com/article/1171206-treatment

  Pharmacologic therapy includes anticholinesterase medication and immunosuppressive agents, such as corticosteroids and nonsteroid immunosuppressants like azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, sirolimus, rituximab, cyclophosphamide, and other immunomodulatory therapies that include plasmapheresis, and intravenous immune globulin (IVIg). […] Plasmapheresis and thymectomy are also employed to treat MG. They are not traditional medical immunomodulating therapies, but they function by modifying the immune system. Thymectomy is an important treatment option for MG, especially if a thymoma is present. […] Intubation and intensive care unit (ICU) transfer usually are reserved for patients in myasthenic crisis with respiratory failure. […] Acetylcholinesterase (AChE) inhibitors and immunomodulating therapies are the mainstays of myasthenia gravis (MG) treatment.

• #9 Myasthenia gravis symptoms, diagnosis & treatment – TeleMed2U

  https://www.telemed2u.com/neurology/myasthenia-gravis

  Monoclonal antibodies target the process that damages or destroys muscle function. […] Anticholinesterase medications treat MG by slowing the breakdown of nerve-to-muscle transmission and increasing muscle strength. […] Corticosteroids provide improvement, or a complete relief of symptoms, in more than 75% of patients. It can cause side effects. If patients dont do well on prednisone, azathioprine may be used instead, or with prednisone. […] Immunosuppressive medications suppress abnormal antibodies. They can cause major side effects and must be monitored carefully. […] Plasmapheresis removes harmful antibodies from your blood plasma and replaces them with normal plasma. Intravenous immunoglobulin gives you a highly concentrated dose of antibodies to temporarily change how the immune system works. Only used in severe cases of MG, this therapy lasts a few weeks to months.

• #10 Treatments for Myasthenia Gravis – Myasthenia Gravis

  https://www.myasthenia.au/background/treatments/

  The need for anticholinesterases varies from day-to-day, and during the same day in response to infection, menstruation, emotional stress, and hot weather. […] Anticholinesterases are the gentlest of the drugs available to treat MG. […] Immunosuppressant drug therapy attempts to suppress the body’s immune system, although no one really knows how they work in MG. […] The immune system produces antibodies to fights against foreign bacteria and viruses. […] Immunosuppressive drugs suppresses the production of antibodies to stabilize an overactive immune system. […] In decreasing order of their frequency of use in MG, they are: prednisone, azathioprine (Imuran), cyclophosphamide (Cytoxan) and cyclosporine (Sandimmune). […] Prednisone is a synthetic drug which resembles natural hormones produced by the cortex of human adrenal glands, and is used to treat many illnesses.

• #10 Treatments for Myasthenia Gravis – Myasthenia Gravis

  https://www.myasthenia.au/background/treatments/

  Unique to MG is the possibility of increasing weakness during the first two weeks of prednisone therapy, which requires close medical supervision when first administered. […] Prednisone has a great many potential undesirable effects, usually related to dose and duration of drug use. […] Azathioprine also suppresses the production of ACh receptor antibodies. […] Cytoxan is considered only for the most severe cases of MG when other therapies have failed. […] Sandimmune is an immunosuppresion used during organ transplantation, and could be of benefit to myasthenics in reducing the dose of prednisone. […] CellCept is a relatively new immunosuppressive drug that was originally developed to prevent immune rejection of transplanted organs. […] Plasmapheresis is a blood purification procedure used to treat several autoimmune diseases.

• #10 Treatments for Myasthenia Gravis – Myasthenia Gravis

  https://www.myasthenia.au/background/treatments/

  Plasmapheresis does not cure MG it only temporarily reduces the level of circuiting antibodies that attack the neuromuscular junction. […] Intravenous immune globin (IVIG) is the opposite of plasmapheresis instead of drawing off the offending antibodies, IVIG swamps the body with pooled gamma globulin antibodies from many donors. […] Thymectomy is the removal of the thymus gland by surgery. […] The goal of thymectomy as a treatment for MG is to induce remission, or at least improvement, permitting a reduction in immunosuppressive medication. […] After a thymectomy, remission or marked improvement occurs in more than half of cases. […] About 30% of MG patients without a thymoma who undergo thymectomy eventually go into complete drug-free remission, and another 50% experience marked improvement.

• #11 Current Treatment of Myasthenia Gravis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8950430/

  Corticosteroids are the first-line immunosuppressant therapy for patients with MG who remain symptomatic while on AChE inhibitors or those who desire better symptom control. […] Azathioprine is one of the most widely used steroid-sparing drugs for MG. […] Tacrolimus is a calcineurin inhibitor that suppresses T-cell activity and proliferation by blocking the transcription of cytokine genes including IL-2, a similar mechanism of action as cyclosporine but with stronger immunosuppressive effects. […] Mycophenolate mofetil suppresses T- and B-lymphocytes proliferation by inhibiting the enzyme inosine monophosphate dehydrogenase involved in the biosynthesis of de novo guanosine nucleotides. […] Cyclophosphamide is non-phase-specific alkylating agent that acts on DNA and inhibits rapidly proliferating cells such as T- and B-lymphocytes.

• #11 Current Treatment of Myasthenia Gravis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8950430/

  Myasthenia gravis (MG) is the most extensively studied antibody-mediated disease in humans. Substantial progress has been made in the treatment of MG in the last century, resulting in a change of its natural course from a disease with poor prognosis with a high mortality rate in the early 20th century to a treatable condition with a large proportion of patients attaining very good disease control. This review summarizes the current treatment options for MG, including non-immunosuppressive and immunosuppressive treatments, as well as thymectomy and targeted immunomodulatory drugs. […] Treatment of MG remains challenging as a subgroup of patients are treatment refractory, therefore having recurrent hospitalizations for MG crisis, requiring maintenance IVIG or PLEX. Therefore, more aggressive approaches such as rebooting of the immune system with high-dose cyclophosphamide or autologous bone marrow transplantation were used in some refractory cases with life-threatening disease.

• #12 Long-Term Patient Outcomes: The Role of Sustained Treatment Efficacy in Myasthenia Gravis – European Medical Journal

  https://www.emjreviews.com/neurology/article/long-term-patient-outcomes-the-role-of-sustained-treatment-efficacy-in-myasthenia-gravis-s110125/

  While this is usually achievable with conventional treatments, around 20% of patients still experience symptoms and QoL impact. […] As per the 2016 consensus paper, first-line therapy is usually cholinesterase inhibitors, such as pyridostigmine: a recommendation that remained unchanged in the documents 2020 update. […] In mild MG, these agents can produce rapid relief of symptoms, but most people will need some form of immunosuppression to suppress autoantibody production. […] Available agents include azathioprine, mycophenolate mofetil, cyclosporin, cyclophosphamide, tacrolimus, and rituximab. […] This approach, however, does not work for everyone, and some patients will still require small doses of corticosteroids to maintain control. […] The problem here is that there are so many side effects.

• #12 Long-Term Patient Outcomes: The Role of Sustained Treatment Efficacy in Myasthenia Gravis – European Medical Journal

  https://www.emjreviews.com/neurology/article/long-term-patient-outcomes-the-role-of-sustained-treatment-efficacy-in-myasthenia-gravis-s110125/

  Despite all these options, the experts highlighted that there is still a subset of patients, up to 20%, who experience symptoms and relapses, despite using conventional treatments. […] There are still patients who are not well controlled, even though they have been treated for years and may have tried three, four, or five treatments. […] Recent years have seen the emergence of novel therapies that could help improve treatment efficacy, potentially with an improved tolerability profile. […] Some of these novel treatments have already been approved and are being used, and I am sure others will be arriving on the market soon, said Claeys. […] These new treatments, however, are not universally available. […] The MG treatment field has come a long way in the last decade or so, but the healthcare community can never be satisfied when there is still a sizable fraction of the patient population that is not well controlled. […] Clinical experience suggests that enhancing symptom management and QoL is what matters to patients with gMG. […] Sustained treatment efficacy, experts are convinced, is key. […] In the future, new therapies could allow for more sustained disease management.

• #13 Myasthenia Gravis Treatments | Myasthenia Gravis Foundation of America

  https://myasthenia.org/myasthenia-gravis-treatments/

  Imuran (azathioprine) is a non-steroidal immunosuppressant that is also used in the treatment of myasthenia gravis. […] CellCept (mycophenolate mofetil) is another non-steroidal immunosuppressant that reduces T-cell and B-cell production. […] Cyclosporin is a type of immunosuppressant called a calcineurin inhibitor. […] Targeted treatments […] Treatments that target B-cells are thought to reduce this immune response and the levels of abnormal antibodies that can cause myasthenia gravis symptoms. […] Rituximab is a monoclonal antibody that targets and attacks B-cells. […] Neonatal Fc receptor blockers attach to and block the neonatal Fc receptor, reducing the levels of immunoglobulin G antibodies, including the abnormal antibodies responsible for myasthenia gravis symptoms. […] Complement inhibitors can reduce activation of the complement pathway by blocking certain complement proteins, including C5.

• #13 Myasthenia Gravis Treatments | Myasthenia Gravis Foundation of America

  https://myasthenia.org/myasthenia-gravis-treatments/

  Soliris (eculizumab) is a monoclonal antibody that inhibits the complement pathway by targeting the C5 complement protein. […] Ultomiris (ravulizumab-cwvz) is another complement inhibitor that targets the C5 complement protein to reduce activation of the complement pathway. […] Zilbrysq (zilucoplan) is approved for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor antibody-positive. […] Intravenous immunoglobulin is used as a short-term treatment in an emergency, if other treatments are not working or when a quick improvement in strength is needed. […] Therapeutic plasma exchange is a treatment that takes your blood and separates the different components to remove the blood plasma that contains the abnormal antibodies responsible for myasthenia gravis symptoms.

• #13 Myasthenia Gravis Treatments | Myasthenia Gravis Foundation of America

  https://myasthenia.org/myasthenia-gravis-treatments/

  Treatment Options: Surgery […] Thymectomy is the surgical removal of the thymus gland. […] Thymectomy can be carried out if you have myasthenia gravis with or without thymus abnormalities. […] The surgery can help to improve your muscle weakness and the number of medications needed or may even provide remission of your symptoms. […] Treatment Options: Medicine […] Acetylcholinesterase inhibitors are often part of the initial treatment for myasthenia gravis, and one of the most used drugs is pyridostigmine bromide. […] Corticosteroids or other immunosuppressants are usually the next lines of treatment if treatment beyond acetylcholinesterase inhibitors is needed. […] Prednisone is a common type of corticosteroid used in the treatment of many conditions including myasthenia gravis.

• #14

  https://link.springer.com/article/10.1007/s40259-024-00701-1

  Standard treatment of MG consists of symptomatic treatment with acetylcholine-esterase-inhibitors and disease-modifying treatment with steroids and steroid-sparing immunosuppressants for long-term management. Patients fulfilling criteria for prognostic importance of thymectomy should undergo thymectomy within the first 2 years after diagnosis. […] Overall, between 50% and 70% of patients with gMG achieve minimal manifestation, pharmacological remission or complete stable remission under standard treatment. The use of steroids is limited by significant adverse effects and the use of standard steroid-sparing immunosuppressants as well as thymectomy show a relevant clinical response only after months or years. Taken together, this highlights the need for better tolerated, more targeted therapies to treat gMG.

• #14

  https://link.springer.com/article/10.1007/s40259-024-00701-1

  The regulatory approval of several new monoclonal antibodies for MG has led to a shift from broad-spectrum immunosuppressants alone to add-on target-specific and selective immunological agents. Generally, MG treatment is becoming more personalised, considering MG subtype and personal patient preferences. […] The availability of more targeted and fast-acting biologicals has raised the hope among patients and clinicians that a substantially larger proportion of patients will achieve satisfactory clinical improvements or even remission status.

• #15 New and emerging treatments for myasthenia gravis | BMJ Medicine

  https://bmjmedicine.bmj.com/content/2/1/e000241

  Myasthenia gravis is an antibody mediated autoimmune disorder affecting the neuromuscular junction function that leads to muscle weakness and functional disability. […] Treatments targeting the underlying pathophysiological pathway are rapidly evolving and have improved management of myasthenia gravis. […] The most appropriate selection of treatments considers the mechanisms, indications, risks and benefits, and costs of each treatment along with patients preference. […] Targeting different pathomechanisms of the disease simultaneously will likely lead to improved outcomes, particularly in the severe cases. […] Although many patients with myasthenia gravis benefit from standard treatments, including cholinesterase inhibitors, corticosteroids, and steroid sparing immunosuppressants (eg, azathioprine and mycophenolate mofetil), 8.5-15% of patients still have varying degrees of disability because of insufficiently controlled clinical symptoms or unacceptable side effects.

• #15 New and emerging treatments for myasthenia gravis | BMJ Medicine

  https://bmjmedicine.bmj.com/content/2/1/e000241

  A large systematic review of 165 patients with AChR+gMG treated with rituximab from 13 different studies reported substantial clinical improvement in 68% of patients (113/165); one study showed a remission rate of 36% (14/39), four studies found that 27-64% (mean 54%) of patients had minimal manifestation status, whereas nine studies reported a significant reduction in immunosuppressive treatment burden. […] The 2020 update of the international myasthenia gravis consensus guidelines recommend consideration of rituximab as an early treatment option for those with MuSK+MG who have had an unsatisfactory response to initial immunotherapy. […] Eculizumab is a recombinant monoclonal antibody that binds to C5, inhibits enzymatic cleavage to C5a and C5b, and ultimately prevents formation of the membrane attack complex.

• #15 New and emerging treatments for myasthenia gravis | BMJ Medicine

  https://bmjmedicine.bmj.com/content/2/1/e000241

  Eculizumab was approved by the US Food and Drug Administration in 2017 for use in AChR+gMG. […] The pivotal phase 3 study, REGAIN (Safety and Efficacy of Eculizumab in Refractory Generalised Myasthenia Gravis), explored the efficacy and safety of eculizumab in refractory AChR+gMG. […] Ravulizumab is a long acting C5 complement inhibitor engineered over the backbone of eculizumab. […] The phase 3 CHAMPION (Safety and Efficacy Study of Ravulizumab in Adults With Generalised Myasthenia Gravis) study evaluating the safety and efficacy of ravulizumab in patients with AChR+gMG showed significant improvement in the total score on the MG-ADL scale (P0.001) and in the quantitative myasthenia gravis score from baseline to week 26 (P0.001) compared with placebo. […] Efgartigimod alfa can be prescribed to eligible patients with generalised myasthenia gravis through the Early Access to Medicines Scheme in the UK. […] Rozanolixizumab was granted FDA approval for the treatment of AChR+gMG in June 2023. […] The guidelines that direct the clinical care of our patients with myasthenia gravis will need to be updated frequently to reflect the rapidly changing tools available to treat these patients.

• #15 New and emerging treatments for myasthenia gravis | BMJ Medicine

  https://bmjmedicine.bmj.com/content/2/1/e000241

  In recent years, research has identified promising new treatments, including targeted treatments with new mechanisms of action that have the potential to improve efficacy and tolerability. […] The international consensus guidance recommends early consideration of thymectomy in patients with AChR antibody positive generalised myasthenia gravis (AChR+gMG), aged 18-50 years, although the guideline from the Association of British Neurologists emphasises early thymectomy in patients with general and ocular myasthenia gravis who are younger than 45 years. […] Our understanding of the pathophysiology of myasthenia gravis has enabled the development of new targeted therapeutics with more favourable side effect profiles. […] Rituximab is a chimeric monoclonal antibody against CD20 that depletes circulating B cells.

• #16 Insights Into the Current and Future Treatment of Myasthenia Gravis

  https://www.neurologylive.com/view/insights-into-the-current-and-future-treatment-of-myasthenia-gravis

  Individuals with ocular disease can be debilitated by it, Silvestri continued, particularly if treatment with cholinesterase inhibitors falls short. In those instances, he said, he turns to steroid treatment. […] I will move on to an early immunosuppressant. I have a couple [patients who] had quite severe ocular myasthenia who are maintained long-term [and are] doing really well on mycophenolate, he said. […] When it comes to the population of patients with MuSK-mediated myasthenia gravis, Silvestri noted that treatment with rituximab (Rituxan; Genentech) is his mainstay approach, as the literature are mostly aligned on the effects of B-cell-depleting therapy in myasthenia gravis. […] Honestly, the [past] few patients I’ve diagnosed with MuSK, I’ve gone fairly early to rituximab and often it takes just 1 cycle to give them a pretty long-lasting response.

• #16 Insights Into the Current and Future Treatment of Myasthenia Gravis

  https://www.neurologylive.com/view/insights-into-the-current-and-future-treatment-of-myasthenia-gravis

  Both experts expressed that high-dose steroid treatment is often undesirable because of growth and behavioral issues. […] I think there’s a role for our newer therapies in these kids, and we’re having success. […] For patients who are pregnant, the pair noted that pyridostigmine, prednisone, and intravenous immunoglobulin are all safe to consider. […] We will use prednisone [because it] does not cross the placental barrier. […] Despite the progress in treatment and the ongoing development in the pipeline, there are lingering needs for those with myasthenia gravis. […] And, certainly, [there’s an unmet need] in patients who don’t respond to any of the therapies that are currently available, Silvestri said. […] In addition, we need more data in our pediatric populations. […] He also said that the European Medicines Agency mandates for small trials in the pediatric populations have added to their knowledge in the past few decades. […] For Howard, the necessity is primarily for safety data during pregnancy. […] [Although] it represents a very small portion of the total population, for your practice and my practice, it represents the bulk of the patients we see because no one knows what to do with them, Howard said.

• #17 Myasthenia Gravis – a new era of treatment options  | ACNR

  https://acnr.co.uk/articles/new-treatment-options-for-myasthenia-gravis/

  Although symptoms in most MG patients are eventually adequately controlled on the current available therapies, over 10% of patients are refractory and real world studies show that over 40% of patients have unacceptable disease control as measured by a Myasthenia Gravis Activities of Daily Living (MG ADL) score greater than three. […] There has therefore been a longstanding need for specific, targeted treatments that are more efficacious, with a faster onset of action. Advances in the understanding of the pathogenesis of MG have unveiled a number of new treatment targets which have the potential to herald a new era for the management of patients with MG. […] The classical complement pathway is implicated in the pathogenesis of AChR MG: formation of the membrane attack complex leads to destruction of the neuromuscular end-plate. Targeting complement is therefore a potential treatment to mitigate the pathogenic effects of AChR antibodies in MG.

• #17 Myasthenia Gravis – a new era of treatment options  | ACNR

  https://acnr.co.uk/articles/new-treatment-options-for-myasthenia-gravis/

  Eculizumab, a humanised anti-C5 monoclonal antibody was the first complement inhibitor trialled in MG and examined its efficacy in patients with refractory generalised MG. […] Ravulizumab is a C5-inhibitor that has been engineered to have an extended half-life and is given as an IV infusion every 8 weeks. […] A further complement inhibitor is Zilucoplan, a 15 amino acid cyclic peptide that inhibits the complement cascade by binding to C5a and C5B with high affinity. […] Blocking of the FC receptor interferes with this recycling and reduces the plasma concentration of IgG. By reducing the level of IgG, the effect of the pathogenic AChR antibodies in MG can be reduced, and thus FcRN blocking can be thought of as similar to ‘medical plasma exchange’. […] Various FcRN blocking agents have been investigated or are currently undergoing Phase 3 trials for the treatment of generalised MG. […] The landscape of MG treatment is clearly changing. Rather than slow acting non-specific treatments we now have more defined targets for treatments with a faster onset of action.

• #18 Myasthenia Gravis Treatment

  https://practicalneurology.com/articles/2020-july-aug/myasthenia-gravis-treatment-1

  The FcRn plays a crucial role in IgG homeostasis by rescuing IgGs from lysosomal degradation and thereby increasing the long half-lives of IgGs compared with other Ig isotypes. […] Autologous hematopoietic stem cell transplantation (HSCT) for treatment of MG has been reported in a few retrospective studies and a phase 2 trial is underway. […] A variety of therapeutics are being developed to target specific immune system functions in contrast to the broader immunosuppressive approach that has traditionally been available.

• #19 Advancements in Myasthenia Gravis Treatment

  https://www.uspharmacist.com/article/advancements-in-myasthenia-gravis-treatment

  Rozanolixizumab (Rystiggo): The newly FDA-approved drug rozanolixizumab (June 2023) is indicated for the treatment of generalized MG (gMG) in adult patients who are positive for antiacetylcholine receptor (AChR) or antimuscle-specific tyrosine kinase (MuSK) antibodies. Rozanolixizumab is a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRn), leading to a reduction in circulating IgG. Its high affinity to FcRn prevents the binding of AChR and MuSK autoantibodies to FcRn, facilitating the removal of these autoantibodies from the body. As a result, these autoantibodies lose their capacity to disrupt neuromuscular signaling, thereby reducing the occurrence of muscle weakness. This mechanism ultimately restores signaling at the neuromuscular junction. […] Zilucoplan (Zilbrysq): In October 2023, the FDA granted approval to zilucoplan, a complement inhibitor designed for the treatment of gMG in adult patients with AChR antibody positivity. Zilucoplan works by binding to the complement protein C5, preventing its cleavage into C5a and C5b. This inhibition prevents the formation of the terminal complement complex, C5b-9, and is presumed to reduce its deposition at the neuromuscular junction. The exact mechanism for the therapeutic effects of this drug in MG remains unknown.

• #20 FDA Approves New Treatment for Myasthenia Gravis | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-myasthenia-gravis

  The U.S. Food and Drug Administration today approved Vyvgart (efgartigimod) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. […] Todays approval is an important step in providing a novel therapy option for patients and underscores the agencys commitment to help make new treatment options available for people living with rare diseases. […] Vyvgart is the first approval of a new class of medication. It is an antibody fragment that binds to the neonatal Fc receptor (FcRn), preventing FcRn from recycling immunoglobulin G (IgG) back into the blood. The medication causes a reduction in overall levels of IgG, including the abnormal AChR antibodies that are present in myasthenia gravis. […] The safety and efficacy of Vyvgart were evaluated in a 26-week clinical study of 167 patients with myasthenia gravis who were randomized to receive either Vyvgart or placebo. The study showed that more patients with myasthenia gravis with antibodies responded to treatment during the first cycle of Vyvgart (68%) compared to those who received placebo (30%) on a measure that assesses the impact of myasthenia gravis on daily function. More patients receiving Vyvgart also demonstrated response on a measure of muscle weakness compared to placebo. […] Patients using Vyvgart should monitor for signs and symptoms of infections during treatment. Health care professionals should administer appropriate treatment and consider delaying administration of Vyvgart to patients with an active infection until the infection is resolved.

• #21 New Myasthenia Gravis treatments 2025 | Everyone.org

  https://everyone.org/explore/treatment/?id=104

  Recent advances in MG treatment include targeted biological therapies such as Vyvgart (efgartigimod alfa), Rystiggo (rozanolixizumab), and Zilbrysq (zilucoplan). […] Vyvgart and Rystiggo target the neonatal Fc receptor (FcRn), reducing pathogenic autoantibodies that cause MG symptoms. […] Zilbrysq inhibits complement activation, reducing autoimmune damage at the neuromuscular junction. […] Thymectomy, the surgical removal of the thymus gland, is a standard treatment option for MG patients, particularly those with generalized MG and thymoma (a tumor of the thymus gland). […] Thymectomy may take several months to years to demonstrate its full therapeutic effect. […] Plasma exchange involves removing plasma containing harmful antibodies from the patient’s blood and replacing it with donor plasma or albumin solution.

• #21 New Myasthenia Gravis treatments 2025 | Everyone.org

  https://everyone.org/explore/treatment/?id=104

  Plasma exchange provides rapid but short-lived improvement in muscle strength. […] IVIG therapy involves administering pooled immunoglobulins intravenously to temporarily modulate the immune system. […] IVIG can rapidly improve muscle strength and function, though its effects are temporary, typically lasting several weeks. […] Emerging therapies and research directions include ongoing research into stem cell therapies and gene therapies. […] These advanced therapeutic strategies aim to reset or modify the immune system, potentially providing long-term remission or even a cure in the future. […] In summary, while no definitive cure currently exists for myasthenia gravis, numerous effective treatment options are available to manage symptoms and improve patients’ quality of life.

• #21 New Myasthenia Gravis treatments 2025 | Everyone.org

  https://everyone.org/explore/treatment/?id=104

  Treatment options for Myasthenia Gravis vary depending on the severity and individual patient needs. Common medications include cholinesterase inhibitors such as pyridostigmine, which improve nerve-muscle communication and reduce symptoms. […] Immunosuppressive therapies like corticosteroids, azathioprine, mycophenolate mofetil, and cyclosporine help control the immune response and minimize muscle weakness. […] In recent years, newer targeted therapies such as eculizumab and ravulizumab have been approved for patients with refractory generalized MG, offering additional options for those who do not respond adequately to conventional treatments. […] Treatment of MG aims to relieve symptoms, improve muscle strength, and reduce autoimmune activity. […] The choice of treatment depends on disease severity, antibody status (anti-AChR or anti-MuSK), patient age, comorbid conditions, and response to previous therapies.

• #22 Myasthenia Gravis | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/myasthenia-gravis

  Currently, there is no known cure for myasthenia gravis. Most people with myasthenia gravis live to an average life expectancy. There are treatments available to help reduce and improve muscle weakness, including: […] Anticholinesterase medications. Medications to treat myasthenia gravis include mestinon or pyridostigmine, which slow the breakdown of acetylcholine at the neuromuscular junction and improve neuromuscular transmission and increase muscle strength. […] Thymectomy. A thymectomy is an operation to remove the thymus gland. It can reduce a persons symptoms, possibly by rebalancing the immune system. […] Complement inhibition. Complement is a collection of naturally produced proteins that function as part of the immune response. […] Immunosuppressive drugs. Immunosuppressive drugs can improve muscle strength by suppressing the production of abnormal antibodies.

• #23 Medical Management – Myasthenia Gravis (MG) – Diseases | Muscular Dystrophy Association

  https://www.mda.org/disease/myasthenia-gravis/medical-management

  Thymectomy surgical removal of the thymus gland is recommended for thymoma and for most cases of generalized MG. […] In plasmapheresis, also known as plasma exchange, an intravenous line is used to remove antibodies from the blood. […] These treatments bring about fast but short-lived relief from MG, and are mostly used until other medications take effect, prior to surgery or for myasthenic crisis. […] When MG is properly treated, crisis is very rare, with a 2% to 3% risk for MG patients. […] Evaluation and management of myasthenic crisis: Admission to intensive care unit, Frequent monitoring of respiratory muscle strength, Elective intubation if clinical evaluation suggests impending respiratory failure, Initiation of rapid therapy with plasma exchange or intravenous immune globulin (IVIG), Initiation of immunomodulating therapy with high-dose glucocorticoids. […] In rare cases, pregnancy appears to trigger the onset of MG.

• #24 Immunotherapy for Myasthenia Gravis | NYU Langone Health

  https://nyulangone.org/conditions/myasthenia-gravis/treatments/immunotherapy-for-myasthenia-gravis

  If symptoms of myasthenia gravis are severe if you’re unable to open your eyes or move your lips, for example your doctor may prescribe a protein called immunoglobulin to be administered through a vein with intravenous (IV) infusion. IV immunoglobulin therapy uses helpful antibodies taken from donor plasma to suppress inflammation. A doctor then administers these donor antibodies into your body through the veins to suppress inflammation. These infusions are given daily for about five days. Most people receive regular maintenance therapy every few weeks over the course of several months to keep the immune system suppressed until symptoms are reduced. IV immunoglobulin therapy can be used along with other treatments or until medications begin working. […] If a flare-up occurs, your doctor may treat you with medication or IV immunoglobulin therapy.

• #25 Myasthenia gravis – Wikipedia

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

  Myasthenia gravis is generally treated with medications known as acetylcholinesterase inhibitors, such as neostigmine and pyridostigmine. […] Immunosuppressants, such as prednisone or azathioprine, may also be used. […] The surgical removal of the thymus may improve symptoms in certain cases. […] Plasmapheresis and high-dose intravenous immunoglobulin may be used during sudden flares of the condition. […] If the breathing muscles become significantly weak, mechanical ventilation may be required. […] Once intubated acetylcholinesterase inhibitors may be temporarily held to reduce airway secretions. […] About 10% of people with generalized MG are considered treatment-refractory. […] Autologous hematopoietic stem cell transplantation (HSCT) is sometimes used in severe, treatment-refractory MG.

• #26 Myasthenia Gravis Treatment With CAR T-Cell Therapy | Kyverna

  https://kyvernatx.com/patients/myasthenia-gravis/

  Standard treatments for patients with MG include cholinesterase inhibitors, corticosteroids, IVIg, plasma exchange, thymectomy, and steroid sparing immunosuppressants, with newer treatments targeting B cell depletion, complement inhibition, and neonatal Fc receptor inhibition; however, up to 15% of patients continue to have disability due to insufficient control of clinical symptoms and unresolved side effects. […] CAR T-cell therapy involves modifying a patients T cells to recognize and remove B cells in the patients body. Kyvernas CD19 CAR T-cell therapy, KYV-101, specifically targets CD19, a protein expressed on the surface of B cells, which is involved in various types of autoimmune diseases. We believe our approach may present a significant advantage over current standard-of-care therapies by aiming to directly deplete B cells and potentially resetting disease-contributing B cells. […] Participation in clinical trials is the best way to access our investigational product. Kyverna may consider requests for expanded access when alternative therapy options have been exhausted.

• #27 Individualized myasthenia gravis treatment based on well-defined disease pathogenesis – touchNEUROLOGY

  https://touchneurology.com/neuroimmunology/conference-hub/individualized-myasthenia-gravis-treatment-based-on-well-defined-disease-pathogenesis/

  Now, to continue with where I finished to your last question, I would say that real-life data comparisons between the various new treatments: the FcRn inhibitors, the complement inhibitors, the B-cell active drugs, anti-CD19, anti-CD20, and others. Real-life comparisons and experience with these new drugs will be very important. […] The aim is, of course, to have much more specific treatment, suppressing the immune response against a acetylcholine receptor or the MuSK antigen, and not all other immune cells in the body. […] The new therapies that are coming up [that] have been published in small patient series and for individual patients is T-cell directed therapies, therapies with chimeric modified T cells against the T cell receptor or against autoimmune antibodies: so called CAR-T therapy. […] That is combination of drugs, but also non-drug therapies: thymectomy, for example, and also education of patients, regarding work working ability.

• #28 Myasthenia Gravis: What It Is, Causes, Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/17252-myasthenia-gravis-mg

  A thymectomy is surgery to remove the thymus gland. […] Common medications to treat myasthenia gravis include: […] They boost signals between nerves and muscles to improve muscle strength. […] Medications like corticosteroids decrease inflammation and reduce your bodys production of abnormal antibodies. […] If you have myasthenia gravis, try these steps to ease fatigue and boost muscle strength: […] Exercise regularly to strengthen muscles, boost your mood and give you more energy. […] Avoid going outside in the middle of a hot day. […] Get plenty of protein and carbohydrates in your meals for added energy. […] Tackle your most exhausting tasks earlier in the day when you feel your best. […] Take naps or rest breaks throughout the day.

• #29 Navigating the Shifting Landscape of Myasthenia Gravis Treatment: The Impact of Expensive Targeted Therapies – IQVIA

  https://www.iqvia.com/library/articles/navigating-the-shifting-landscape-of-myasthenia-gravis-treatment

  The current market leader acts by inhibiting the complement system but is losing its US patent in 2027. It is expected to lose its position to a drug which acts by inhibiting the neonatal Fc receptor (FcRn) and offers better efficacy without any boxed warning. Before 2017, there was a significant unmet need for targeted therapies as only symptomatic treatments were available for patients suffering from MG. The landscape of MG treatment is set to change significantly, with new drugs targeting intricate nuances of the body’s immune response.

• #30 Myasthenia Gravis: New Drugs and a Road to Individualized Treatment > News > Yale Medicine

  https://www.yalemedicine.org/news/myasthenia-gravis-new-drugs-individualized-treatment

  Both OConnor and Dr. Nowak hope to see a continuing paradigm shift in myasthenia gravis treatment in the next seven to ten years based on research. […] For most patients, myasthenia gravis can now be considered a chronic disease that is treatable with the right individual treatment strategymaking the condition much less threatening than it was even five to 10 years ago, Dr. Nowak explains.

• #31 How is treatment for myasthenia gravis evolving? – Harvard Health

  https://www.health.harvard.edu/blog/how-is-treatment-for-myasthenia-gravis-evolving-2021041222358

  Many people with autoimmune MG need long-term immunosuppressive treatment to help them manage their disorder. The best options for treatment are decided through monitoring the course of the disease and response to treatments over time. […] The treatment of MG is complex. Your neurology team will take into account several factors, including age, gender, pregnancy and breastfeeding, and other illnesses (such as high blood pressure, diabetes, or liver disease). Theyll also consider the presence and type of MG antibodies, the type of MG youre experiencing (ocular MG or generalized MG, which affects more than the eyes alone), and your preferences and values. […] As new information about treatment becomes available, discuss possible treatment changes with your neurologist, who can help ensure that the treatment choices you make align with your goals for improvement, as well as your personal values and preferences.

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