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Myasthenia Gravis Learning Zone

Navigating myasthenia gravis care

Read time: 40 mins
Last updated: 1st Oct 2025
Published: 15th Aug 2023
Declaration of sponsorship: argenx
Myasthenia Gravis – Overview

Myasthenia gravis epidemiology and pathophysiology

Overview

Myasthenia gravis (MG) is a rare, chronic, B-cell-mediated autoimmune disease and the most prevalent primary disorder affecting neuromuscular transmission. People with MG develop antibodies against transmembrane proteins of the neuromuscular junction, most commonly the acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK)1. This results in fluctuating muscle weakness and fatigue, which can be debilitating, and people with the condition may also experience myasthenic crises, in which rapid and severe worsening of symptoms can be life-threatening1,2.

Over the past 2 decades, significant progress has been made in unravelling the pathophysiology and immunopathology of MG, propelling it to the forefront of autoimmune disease research. What was once an enigmatic condition confined to the purview of neurologists has now transformed into a well-understood entity3.

Timely diagnosis and appropriate treatment of this predominantly treatable disease are crucial for preventing significant morbidity and mortality4. MG is a heterogeneous condition in terms of both phenotype and pathogenesis. The spectrum of symptoms ranges from isolated ocular manifestations to profound weakness affecting the limbs, bulbar region and respiratory muscles.

MG is considered a prototypical antibody-mediated autoimmune disease5. The majority of people with MG present with autoantibodies directed against acetylcholine receptors (AChRs), while a minority exhibit seropositivity for MuSK antibodies, low-density lipoprotein receptor-related protein 4 (LRP4) antibodies or agrin antibodies6-9.

Autoantibodies in MG serve as the basis for defining disease subgroups and delineating phenotypic variations. In a subgroup of people with MG, striational antibodies have also been identified, targeting titin, ryanodine receptor, and the alpha subunit of the voltage-gated K+ channel (Kv1.4)10,11. These antibodies function primarily as biomarkers of disease severity and are often detected in people with late-onset MG or thymoma-associated MG11,12.

Epidemiology

Both the incidence and prevalence of MG have significant geographical variations, but it is believed that incidence has increased worldwide over the past 7 decades3. This increase can be attributed, to some extent, to improvements in the recognition and diagnosis of MG3.

Within Europe, there is wide variation in the incidence and prevalence of MG according to the specific country, the data source and the study date. The reported nationwide incidence rates per million person–years range from 0.17 in Slovakia in 1977–2015 to 29.0 in Sweden in 2006–2016. Nationwide prevalence per 100,000 people ranges from 10.7 in Belarus in 2012 to 36.1 in Sweden in 2006–2016, potentially equating to as many as 123,000 patients in Europe13.

In the United States, the prevalence of MG is estimated to range from 14 to 20 per 100,000 population, amounting to approximately 36,000–60,000 cases. Nevertheless, MG remains underdiagnosed and the actual prevalence is presumed to be higher3,14,15.

Notably, lower incidence and prevalence rates were observed in a comprehensive study conducted in China, with rates reported at 0.155–0.366 per million and 2.19–11.07 per 100,000, respectively16. However, in Korea, two population-based studies indicated a prevalence of 9.67–10.42 per 100,000 individuals in 2010, which increased to 12.99 per 100,000 in 201417.

Over the past 50 years, global prevalence of MG has steadily increased, ranging from 150 to 200 cases per million, in line with increased recognition of the condition18

MG incidence in women shows two peaks around ages 30 and 50 years, while in men it steadily increases with age, peaking between the ages of 60 and 89 years19. Early-onset MG is more common in women, with a female-to-male ratio of 3:1, equalising in the fifth decade13. After age 50 years, prevalence is slightly higher in men, with a male-to-female ratio of 3:27.

MG affects all races; however, it has been shown to be slightly more prevalent in people of African ancestry3. Phenotypic presentations vary across ethnic backgrounds: Black people experience more treatment-resistant ophthalmoplegia and ptosis, while Caucasians experience treatment-refractory generalised MG20. Caucasians have an older age at diagnosis than non-Caucasians, while African Americans have earlier onset and more severe MG, with higher MuSK seropositivity21.

There is evidence for a genetic component to MG. In 35% of cases where one monozygotic twin had MG, so too did the other, whereas the reported familial rate was around 4–7%22. From these figures, the authors calculated that approximately one in every 5,240 people is genetically disposed to MG and that more than half of these individuals will encounter an environmental trigger sufficient to initiate the disease process22.

Pathophysiology

At the neuromuscular junction, ACh is released in discrete quanta from the motor nerve terminal (Figure 1). Subsequently, ACh diffuses across the synaptic cleft and binds to receptors located on the folded membrane of the motor end plate. This binding event initiates depolarisation of the motor end plate and the surrounding muscle membrane. Ultimately, this cascade culminates in the contraction of the muscle fibres23.

LRP4 is a membrane protein that serves as a receptor for agrin in the neuromuscular junction. The binding of agrin to LRP4 activates MuSK, leading to cortactin phosphorylation, which facilitates the clustering of AChR. This clustering enhances receptor availability for synaptic ACh, promoting muscle excitability24.

Pathological process of myasthenia gravis at a postsynaptic receptor at the neuromuscular junction

Figure 1. Pathological process of myasthenia gravis at a postsynaptic receptor at the neuromuscular junction (Adapted25). ACh, acetylcholine; AChR, acetylcholine receptor; IgG, immunoglobulin G; LRP4, low-density lipoprotein receptor-related protein 4; MuSK, muscle-specific kinase.

Myasthenia gravis is characterised by the production of autoantibodies that target the neuromuscular junction (Figure 1). Approximately 85% of people with generalised MG have autoantibodies against the nicotinic AChR. The remaining 15% of people have other autoantibodies, such as anti-MuSK or anti-LRP4. A small number of people with MG have no detectable antibodies to known antigens5,26.

Anti-AChR antibodies – predominantly of the IgG1, IgG2 and IgG3 subclasses (Figure 1) derived from long-lived plasma cells – are produced by B cells in the hyperplastic thymus and other tissue compartments, including the bone marrow27,28. These autoantibodies exert their pathological effects at the neuromuscular junction through various mechanisms. They can block AChR either at, or in the vicinity of, the receptor, inhibiting ACh binding. They can also cross-link AChR by binding to both of its binding sites, resulting in internalisation of the receptor and a reduction in receptor numbers at the neuromuscular junction29.

Additionally, autoantibodies can activate the complement pathway, leading to the formation of the membrane attack complex. Complement-mediated damage results in diminished postsynaptic junctional folds, loss of AChR clustering, voltage-gated sodium channel removal from the membrane and increased synaptic distance29,30.

While our understanding of the pathophysiology of MG mediated by antibodies against AChR and MuSK is relatively comprehensive, less is known about seronegative MG or the role of anti-LRP4 antibodies in MG5,8,30

Myasthenia gravis diagnosis

Symptomatology

Recognising early symptoms of MG and earlier diagnosis can lead to earlier intervention, significantly improving patient outcomes and quality of life40. Professor Heinz Wiendl provides expert insight into early recognition and diagnosis of MG.

 

Myasthenia gravis (MG) is characterised by a wide range of symptoms that primarily affect skeletal muscle function (Figure 2)26. The hallmark feature of MG is muscle weakness that typically worsens with activity and improves with rest7. The specific pattern and severity of symptoms can vary widely among individuals, making MG a complex and heterogeneous disorder31.

Body areas affected by MG

Figure 2. Body areas affected by MG (Adapted32).

Muscle weakness often starts in the muscles that control eye movements, leading to ptosis and diplopia. Weakness in the muscles responsible for facial expressions, swallowing and speaking may also occur, resulting in difficulties with facial movements, chewing and articulation7,33,34. In addition to ocular and facial muscle weakness, MG can also affect other skeletal muscles throughout the body13,35. This can lead to generalised muscle weakness, making it challenging to perform tasks that require physical effort, such as lifting objects, walking, climbing stairs, or even breathing36.

The symptoms of MG can fluctuate over time, with periods of myasthenic crises and periods of relative stability. Factors such as illness, stress, heat and certain medications can trigger or worsen symptoms37.

The following symptoms are commonly associated with myasthenia gravis31,32,38,39:

  • Ocular myasthenia
  • Ptosis
  • Diplopia
  • Changes in facial expressions
  • Dysphagia
  • Shortness of breath
  • Dysarthria
  • Weakness in the arms, hands, fingers, legs and neck

In a 2023 study32, participants were requested to indicate the three symptoms or body regions that troubled them the most. They were required to specify a symptom, a body region, or both when identifying the experiences that caused the greatest distress (Figure 3).

Most distressing MG symptoms and affected body regions, as reported by 28 study participants

Figure 3. Most distressing MG symptoms and affected body regions, as reported by 28 study participants (Adapted32).

Early diagnosis

Early diagnosis of MG is crucial for prompt initiation of appropriate interventions, leading to improved patient outcomes and enhanced quality of life40.

By identifying MG in its early stages, healthcare providers can:

  • differentiate MG from other conditions that may present with similar symptoms, such as Lambert–Eaton myasthenic syndrome or motor neuron diseases38
  • avoid unnecessary investigations and treatments and ensure that specific management strategies for MG are implemented promptly40
  • intervene before the disease progresses and symptoms become more severe40
  • facilitate the timely initiation of disease-modifying therapies41
  • enable monitoring of the disease course more effectively and detect any complications that may arise35

As MG is associated with various complications, such as myasthenic crisis and thymoma42, early detection and management are crucial and can be achieved by undertaking the following diagnostic tests8,38,43:

  • Physical and neurological examination:
    • Muscle strength and tone
    •  Coordination
    • Sense of touch
    • Any impairment of eye movements
  • Edrophonium test
  • Serological testing for:
    • AChR antibodies
    • MuSK antibodies
    • LRP4 antibodies
  • Electrodiagnostics
  • Diagnostic imaging

Diagnostic work-up should also consider medications that may precipitate or exacerbate MG, such as immune checkpoint inhibitors (see Table 1).

Table 1. Medications to be used with caution in MG39. The table lists treatments reportedly associated with worsening of MG symptoms, but it is important to note that some associations may be coincidental. ACh, acetylcholine; MG, myasthenia gravis; FDA, U.S. Food and Drug Administration.

Treatment Notes
Aminoglycoside antibiotics  
Beta-blockers  
Botulinum toxin Should be avoided
Chloroquine/hydroxychloroquine May trigger de novo MG or worsen existing symptoms
Corticosteroids Monitor carefully in first 2 weeks for transient worsening of symptoms
Desferrioxamine (deferoxamine)  
ᴅ-Penicillamine Believed to trigger new MG and should be avoided
Fluoroquinolone antibiotics Carry FDA black box warning for worsening MG. Avoid or use with caution
Immune checkpoint inhibitors May trigger de novo MG or worsen existing symptoms, but important cancer treatments. Use with caution if needed
Iodinated radiological contrast agents Older agents reportedly worsen MG symptoms; newer agents may carry less risk. Use with caution
Macrolide antibiotics  
Magnesium Potentially high risk if given intravenously. Avoid use if possible
Procainamide  
Quinine  
Statins May occasionally trigger or worsen MG
Telethromycin Carries black-box FDA warning for severe, often fatal MG worsening. Should not be used
Live-attenuated vaccines Contraindicated in patients taking immunosuppressive treatments

Current treatment options for myasthenia gravis

Current treatment options

The contemporary treatment landscape is complex – Professor Wiendl provides guidance for fellow physicians to better navigate this everchanging landscape.

 

International consensus guidance on the treatment of myasthenia gravis (MG) has been provided by a panel of 15 international experts assembled at the request of the Myasthenia Gravis Foundation of America (MGFA). The first set of recommendations were published in 201635, with an update in 202039; they provide advice on treatment goals, medications and managing MG in specific subgroups.

The treatment approach for MG is tailored to each individual on the basis of disease severity, response to therapy and potential side effects. Close monitoring, regular follow-up and multidisciplinary care involving neurologists, immunologists and other healthcare professionals are crucial for effectively managing MG and optimising patient outcomes44-46.

There is a range of treatments available for MG but determining the appropriate treatment for each patient is a complex task. Professor Wiendl covers the current treatment options available to people with MG.

 

Treatment goals

The aim of treatment as outlined in the original consensus statement35 is to achieve MGFA Task Force Post-Intervention Status classification Minimal Manifestation Status, where the patient has no symptoms or functional limitations but may have some remaining muscle weakness.

Recommended treatments

As outlined in Figure 4, pyridostigmine is the recommended first-line treatment for the majority of people with MG, with corticosteroids introduced if required and non-steroidal immunosuppressants added if corticosteroids are insufficient or there are contraindications or other issues35.

The core treatment recommendations for myasthenia gravis as outlined in the international consensus guidance

Figure 4: The core treatment recommendations for myasthenia gravis as outlined in the international consensus guidance35.

  • Pyridostigmine
    Pyridostigmine, an acetylcholinesterase (AChE) inhibitor, prevents metabolism of acetylcholine (ACh), thereby increasing its bioavailability at the synaptic cleft, which may help improve muscle weakness47.
  • Corticosteroids
    Despite the longstanding use of prednisone in MG treatment, there is insufficient evidence from controlled studies to firmly establish its effectiveness. However, the EPITOME trial demonstrated a notable superiority of prednisone over placebo specifically in patients with ocular MG39,48,49.
  • Non-steroidal immunosuppressive therapies
    These are used as an alternative to corticosteroids when required, and as a means of reducing exposure to steroids. The 2016 guidelines note ‘widespread variation in practice’ when it comes to the choice of non-steroidal immunosuppressive therapies because of the paucity of evidence supporting one over another35.

Azathioprine and cyclosporine both have supporting evidence from randomised clinical trials, although use of the latter is limited by the risk of side effects, notably hypertension and nephrotoxicity, and it is used only off label50. Cyclophosphamide also has some evidence in its favour, particularly in refractory cases, but is again limited by side effects and does not have regulatory approval for MG51-53. The other options have little or no supporting evidence yet are often used off label and are even recommended in national guidelines35.

Treatments for refractory MG and myasthenic crisis

Intravenous therapies

Plasma-exchange and intravenous immunoglobulins are recommended as short-term treatments in the following situations35:

  • Life-threatening complications, such as respiratory insufficiency
  • Patients with significant bulbar dysfunction preparing for surgery
  • When rapid symptom improvement is required
  • For severe symptoms refractory to other treatments
  • To prevent/minimise symptom worsening that can occur during initiation of corticosteroids

Studies indicate that plasma exchange is effective in about 60–70% of people treated, with improvement closely tied to plasma removal timing. These treatments are mainly indicated for acute worsening of the disease, such as severe generalised or bulbar symptoms, or during a myasthenic crisis54. However, the consensus guidance also suggests use of intravenous immunoglobulin as maintenance therapy in patients with refractory MG or if other options are contraindicated.

Complex MG can pose additional challenges for patients and physicians. Professor Wiendl provides insight into treatment and treatment selection for patients with complicated MG.

 

Approved biologic therapies

Eculizumab

A multicentre phase 3 trial (REGAIN) investigated the use of eculizumab, a monoclonal antibody that targets complement C5, in patients with AChR antibody-positive refractory generalised MG55. The trial did not meet the primary endpoint of a change in MG Activities of Daily Living (MG-ADL) from baseline to week 26, but did meet several of the secondary endpoints, supporting a significant improvement in this patient population55. An open-label extension of REGAIN showed that improvements were maintained for almost 90% of people who remained in the study for 130 weeks and nearly 60% achieved minimal manifestation status56. Eculizumab was approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2017 for treatment of adults with anti-AChR-positive generalised MG57,58. Close monitoring is necessary because of the risk of serious infections associated with eculizumab, as highlighted by the FDA warning58.

Ravulizumab

The phase 3 CHAMPION MG trial evaluated the use of the complement C5 monoclonal antibody ravulizumab in patients with AChR antibody-positive generalised MG59. The trial met the primary endpoint of a change in MG-ADL from baseline to week 26, as well as secondary endpoints, showing significant improvement in this patient population. Ravulizumab received FDA and EMA approval in 2022 for treatment of adults with anti-AChR-positive generalised MG60,61.

Efgartigimod

Efgartigimod is a neonatal Fc receptor (FcRn) antagonist that specifically targets and reduces the level of IgG antibodies. The medication, delivered as a weekly intravenous infusion, was fully approved in 2021 by the FDA and in 2022 by the EMA for treatment of adult patients with generalised MG62,63. A formulation delivered as a weekly subcutaneous injection received FDA approval in June 202364.

Clinical trials, such as the phase 3 ADAPT trial, have shown rapid clinical improvements with efgartigimod – within the first week after administration – leading to reduced disease severity and improved muscle strength65-67. This resulted in enhanced functional abilities and improved quality of life for people with MG68.

Efgartigimod has demonstrated a favourable safety profile, with mostly mild-to-moderate adverse effects, such as injection-site reactions67.

Rozanolixizumab

Rozanolixizumab, a neonatal Fc receptor blocker, suppresses activation of the complement cascade, which plays a crucial role in the pathogenesis of MG66,69. It was approved by the FDA in mid-2023, at which time it was under review by the EMA70,71.

Rozanolixizumab has demonstrated effectiveness across a broad range of MG subtypes, including AChR antibody-positive and MuSK antibody-positive MG72. Like efgartigimod, rozanolixizumab has a rapid onset of action; participants of the MycarinG randomised, double-blind, placebo-controlled, adaptive phase 3 trial had significant improvements in their symptoms by the day 8 study visit72. This response can provide quick relief for people with MG, especially those with severe or refractory MG, and improve their overall quality of life69.

Clinical trials have reported a low incidence of serious adverse events, with the most common side effects being mild-to-moderate infusion-related reactions69,73,74.

Surgical therapy

Thymectomy has been a widely practised treatment for MG for many years, used to minimise or avoid the need for extended immunosuppression. Because of the slow onset of effect, it is an elective procedure, to be performed in stable patients. In a randomised trial published in 2016, 67% of people achieved minimal manifestation status after 36 months compared with 47% in the control group75.

Treatment for MG with MuSK antibodies

This is the one subgroup for whom pyridostigmine may not be the most appropriate first-line treatment; these patients usually have a poor response with a higher than usual rate of side effects35.

The international guidance therefore recommends corticosteroids and/or steroid-sparing immunosuppressive agents, noting that people with MuSK antibodies often require both. Plasma exchange may be helpful, and the 2016 international consensus guidance and 2020 update recommend the anti-CD20 monoclonal antibody rituximab as an early option if response to initial immunotherapy is poor, although it does not have regulatory approval for this indication35,39. Researchers have reported positive results for rituximab in this patient subgroup in small studies76,77, despite its lack of efficacy in phase 2 trials conducted predominantly in those with AChR antibodies78,79.

Ocular MG

The advice for treating ocular MG as outlined in the 2020 update is similar to that for MG overall, with corticosteroids and/or non-steroid immunosuppressive agents advised in patients with functionally limiting or troublesome symptoms that do not respond to pyridostigmine39. Thymectomy may also be considered.

Professor Wiendl provides a closer look at advancements in treatment for MG and the potential benefits they may convey to patients with MG.

 

Unmet needs for myasthenia gravis

The unmet needs of those with MG are complex and at times poorly understood. Professor Wiendl provides insight into those unmet needs and greater insight into how novel and future treatment options may address them.

 

Unknown origin of immunogenicity

Despite advancements in understanding the immunopathology and treatment of MG in recent years, there are still several unmet needs. The origin of immunogenicity remains unknown, with approximately 10% of people with MG testing seronegative for antibodies against AChR, MuSK and LRP480,81. Anti-titin and anti-agrin antibodies have been identified as biomarkers for these seronegative forms, but their clinical significance is not yet well understood82. Studies have shown that anti-titin antibodies were present in 13.4% of triple seronegative people with MG, with implications for disease severity and symptoms82. There is also evidence that anti-titin antibodies are associated with specific forms of MG, affecting clinical outcomes81. Further research is necessary to explore the role of the thymus and the efficacy of thymectomy in MG treatment.

Role of the thymus and thymectomy

The role of the thymus in MG pathophysiology and disease progression remains unresolved83. In some cases, hyperplasia or thymoma may be consequences rather than pathogenic elements. The efficacy of thymectomy has been explored in non-thymomatous MG, and the first randomised clinical trial in this area favoured thymectomy combined with alternate-day prednisone over prednisone alone75. However, further clinical trials are required to identify the subset of people who would benefit most from thymectomy.

Limited evidence for non-steroidal immunosuppressants

Current medical treatment options for MG include cholinesterase inhibitors, corticosteroids and non-steroidal immunosuppressants. However, there is little high-quality evidence supporting non-steroidal immunosuppressants, and nothing comparing these treatments35. Registries, such as the Myasthenia Gravis Foundation of America global MG Patient Registry, could provide valuable data about the effectiveness and safety of these medications in the absence of clinical trials84.

Persistent symptoms and poor quality of life despite treatment

An additional issue is that, despite the availability of multiple treatment options, people with MG continue to experience symptoms and a poor quality of life14,85-87, particularly those with refractory disease4. This highlights the need both for novel therapeutic approaches and stronger data to support decisions about current treatments.

Diagnostic delays and misdiagnosis

Diagnostic delays and misdiagnosis are common, with studies reporting an average time to diagnosis of nearly 1 year88,89. These delays are often due to symptom variability and misinterpretation of symptoms as other conditions, such as stroke or motor neuron disease89.

Lack of standardised disease monitoring tools

Diagnostic tests, like single-fibre electromyography (SFEMG), responsive neurostimulation (RNS), and antibody assays, vary in sensitivity and specificity, further complicating longitudinal monitoring90. This variability limits the ability to personalise care and hampers consistent evaluation across patient populations.

Limited real-world data on emerging therapies

While real-world data (RWD) are emerging for new biologic therapies, such as eculizumab, ravulizumab, and efgartigimod, data for rozanolixizumab and zilucoplan are still scarce91. Moreover, comparisons between therapies are complicated by heterogeneous study designs, endpoints, and sample sizes, making it difficult to draw definitive conclusions for clinical decision-making and emphasising the need for further RWD and comparative studies to guide individualised treatment strategies and assess long-term safety and efficacy91.

Mental health challenges

Beyond clinical gaps, patient-centred and systemic unmet needs remain insufficiently addressed. Fatigue affects approximately 80% of people with MG and is strongly associated with disease severity, depression, anxiety, and sleep disturbances92. It is distinct from muscle fatigability and includes both physical and mental exhaustion92. Cognitive fatigue, though less common, is also reported and may be secondary to depression92,93. Despite its high prevalence, fatigue remains poorly understood and inadequately managed in clinical practice92. People with MG are vulnerable to psychological distress, including depression and anxiety, because of the chronic nature of the disease and its impact on identity and daily functioning94.

Paediatric and older populations

Clinical research involving paediatric and older patients presents distinct challenges, largely due to limited age-specific data and underrepresentation in clinical trials95,96. Juvenile MG, for instance, is rare and lacks randomised controlled trials (RCTs); treatment approaches are often extrapolated from adult population data95. The paediatric population is biologically diverse – prepubertal children differ significantly from postpubertal adolescents – making unified trial designs complex97. Moreover, children cannot provide legal consent, complicating enrolment, and paediatric RCTs are frequently underfunded, as industry efforts tend to prioritise adult populations97. Research access is further restricted by the concentration of care for chronic paediatric conditions in specialised centres97.

Similarly, older patients are more vulnerable to iatrogenic harm, including life-threatening infections linked to immunosuppressive therapies. They are often excluded from clinical trials due to higher rates of treatment-related adverse events, which are commonly driven by polypharmacy, immunosenescence, and comorbidities96,98,99.

Access and affordability of biologics

Standard treatments fail to achieve adequate disease control in approximately 30–50% of patients, driving the need for more effective options91. However, the high cost of biologics raises concerns about cost effectiveness and limits access in many regions, and affordability continues to be a major barrier to equitable delivery91.

Absence of validated biomarkers

The lack of validated biomarkers continues to hinder precision medicine in MG, and although metabolomic profiling shows promise, its clinical utility remains limited at this stage100. Despite advances in targeted therapies, reliable biomarkers for predicting treatment response or guiding therapy selection are currently unavailable91. Clinicians urgently need predictive tools to personalise care and improve clinical outcomes in MG91.

Novel therapies for myasthenia gravis

Professor Wiendl discusses emerging therapies in development for the treatment of MG and how refractory and difficult to treat MG is underrepresented in current clinical trials. Professor Wiendl also discusses the need for biomarkers that will allow physicians to better monitor and treat MG.

 

Zilucoplan

Zilucoplan is another complement C5 inhibitor, which was submitted for regulatory review in November 2022101. It is self-administered by subcutaneous injection, distinguishing it from most other approved and pipeline biological therapies, which require patients to attend a clinic for an intravenous or subcutaneous infusion102.

In the phase 3 RAISE trial, zilucoplan treatment resulted in significant improvements in the primary outcome of change in MG Activities of Daily Living score at week 12, compared with placebo, in a study population of people with generalised MG. About half of this population had refractory disease. Again, these improvements were rapid, with significant separation from placebo occurring by the week 1 study visit103.

The most frequent adverse events in the zilucoplan group were injection-site bruising or pain, diarrhoea and lipase increase.

Batoclimab

Batoclimab is a fully human monoclonal antibody targeting FcRn, designed to reduce circulating pathogenic immunoglobulin (Ig)G antibodies through subcutaneous administration104. In a phase 2a randomised, placebo-controlled trial, batoclimab demonstrated significant reductions in total IgG and anti-acetylcholine receptor (AChR) antibody levels over 6 weeks, with a favourable safety and tolerability profile104. A subsequent press release reported positive topline results from a phase 3 study, where batoclimab achieved clinically meaningful improvements in MG Activities of Daily Living (MG-ADL) scores in AChR-positive patients, with deeper IgG reductions correlating with better clinical outcomes105.

Cemdisiran

Cemdisiran is an investigational small interfering RNA (siRNA) therapy that suppresses hepatic production of complement component C5, a key driver of neuromuscular junction damage in MG106-108.

In the phase 3 NIMBLE trial, subcutaneous cemdisiran every 12 weeks significantly improved MG-ADL scores in patients with generalised MG, meeting both primary and secondary endpoints107,108. When combined with pozelimab (cemdi-poze), cemdisiran achieved near-complete complement inhibition; however, cemdisiran monotherapy demonstrated comparable efficacy with a more convenient dosing schedule107,108.

Cemdisiran was well tolerated, and its quarterly administration may offer practical advantages for long-term disease management107,108.

The cemdisiran manufacturer plans to submit cemdisiran for US regulatory approval in 2026, with additional efficacy data from the ACCESS-1 trial in paroxysmal nocturnal haemoglobinuria (PNH) supporting the submission107,108.

Join our panel of experts in myasthenia gravis as they discuss a range of medical and clinical challenges related to MG management on our Unlocking Myasthenia Gravis podcast channel

References

  1. Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol. 2015;14(10):1023-1036.
  2. Wendell LC, Levine JM. Myasthenic crisis. Neurohospitalist. 2011;1(1):16-22.
  3. Dresser L, Wlodarski R, Rezania K, Soliven B. Myasthenia gravis: epidemiology, pathophysiology and clinical manifestations. Journal of clinical medicine. 2021;10(11):2235.
  4. Schneider-Gold C, Hagenacker T, Melzer N, Ruck T. Understanding the burden of refractory myasthenia gravis. Ther Adv Neurol Disord. 2019;12:1756286419832242.
  5. Fichtner ML, Jiang R, Bourke A, Nowak RJ, O'Connor KC. Autoimmune Pathology in Myasthenia Gravis Disease Subtypes Is Governed by Divergent Mechanisms of Immunopathology. Front Immunol. 2020;11:776.
  6. Bajwa SF, Mohammed RH. Type II Hypersensitivity Reaction. In: StatPearls [Internet]: StatPearls Publishing; 2022.
  7. Ciafaloni E. Myasthenia gravis and congenital myasthenic syndromes. Continuum (Minneap Minn). 2019;25(6):1767-1784.
  8. Lazaridis K, Tzartos SJ. Autoantibody Specificities in Myasthenia Gravis; Implications for Improved Diagnostics and Therapeutics. Front Immunol. 2020;11:212.
  9. Paz ML, Barrantes FJ. Autoimmune Attack of the Neuromuscular Junction in Myasthenia Gravis: Nicotinic Acetylcholine Receptors and Other Targets. ACS Chem Neurosci. 2019;10(5):2186-2194.
  10. Lazaridis K, Tzartos SJ. Myasthenia gravis: autoantibody specificities and their role in MG management. Front Neurol. 2020;11:596981.
  11. Oh SJ. Neuromuscular junction disorders beyond myasthenia gravis. Curr Opin Neurol. 2021;34(5):648-657.
  12. Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev Clin Immunol. 2012;8(5):427-438.
  13. Bubuioc AM, Kudebayeva A, Turuspekova S, Lisnic V, Leone MA. The epidemiology of myasthenia gravis. J Med Life. 2021;14(1):7-16.
  14. Lehnerer S, Jacobi J, Schilling R, Grittner U, Marbin D, Gerischer L, et al. Burden of disease in myasthenia gravis: taking the patient’s perspective. J Neurol. 2022;269(6):3050-3063.
  15. Rath J, Brunner I, Tomschik M, Zulehner G, Hilger E, Krenn M, et al. Frequency and clinical features of treatment-refractory myasthenia gravis. J Neurol. 2020;267(4):1004-1011.
  16. Fang W, Li Y, Mo R, Wang J, Qiu L, Ou C, et al. Hospital and healthcare insurance system record-based epidemiological study of myasthenia gravis in southern and northern China. Neurol Sci. 2020;41(5):1211-1223.
  17. Park SY, Lee JY, Lim NG, Hong YH. Incidence and Prevalence of Myasthenia Gravis in Korea: A Population-Based Study Using the National Health Insurance Claims Database. J Clin Neurol. 2016;12(3):340-344.
  18. Phillips LH, 2nd. The epidemiology of myasthenia gravis. Ann N Y Acad Sci. 2003;998(1):407-412.
  19. Bettini M, Chaves M, Cristiano E, Pagotto V, Perez L, Giunta D, et al. Incidence of autoimmune myasthenia gravis in a health maintenance organization in Buenos Aires, Argentina. Neuroepidemiology. 2017;48(3-4):119-123.
  20. Heckmann JM, Owen EP, Little F. Myasthenia gravis in South Africans: racial differences in clinical manifestations. Neuromuscul Disord. 2007;17(11-12):929-934.
  21. Oh SJ, Morgan MB, Lu L, Hatanaka Y, Hemmi S, Young A, et al. Racial differences in myasthenia gravis in Alabama. Muscle Nerve. 2009;39(3):328-332.
  22. Ramanujam R, Pirskanen R, Ramanujam S, Hammarström L. Utilizing twins concordance rates to infer the predisposition to myasthenia gravis. Twin Res Hum Genet. 2011;14(2):129-136.
  23. Koneczny I, Herbst R. Myasthenia gravis: pathogenic effects of autoantibodies on neuromuscular architecture. Cells. 2019;8(7):671.
  24. Zong Y, Jin R. Structural mechanisms of the agrin-LRP4-MuSK signaling pathway in neuromuscular junction differentiation. Cell Mol Life Sci. 2013;70(17):3077-3088.
  25. Kuks J. Immunopathology of myasthenia gravis. In: Myasthenia Gravis: CRC Press; 2019. p. 147-202.
  26. Behbehani R. Ocular myasthenia gravis: A current overview. Eye Brain. 2023;15:1-13.
  27. Berrih-Aknin S, Le Panse R. [Myasthenia gravis and autoantibodies: Pathophysiology of the different subtypes]. Rev Med Interne. 2014;35(7):413-420.
  28. Leprince C, Cohen-Kaminsky S, Berrih-Aknin S, Vernet-Der Garabedian B, Treton D, Galanaud P, et al. Thymic B cells from myasthenia gravis patients are activated B cells. Phenotypic and functional analysis. J Immunol. 1990;145(7):2115-2122.
  29. Hara H, Hayashi K, Ohta K, Itoh N, Nishitani H, Ohta M. Detection and characterization of blocking-type anti-acetylcholine receptor antibodies in sera from patients with myasthenia gravis. Clin Chem. 1993;39(10):2053-2057.
  30. Higuchi O, Hamuro J, Motomura M, Yamanashi Y. Autoantibodies to low-density lipoprotein receptor-related protein 4 in myasthenia gravis. Ann Neurol. 2011;69(2):418-422.
  31. Regnault A, Morel T, de la Loge C, Mazerolle F, Kaminski HJ, Habib AA. Measuring Overall Severity of Myasthenia Gravis (MG): Evidence for the Added Value of the MG Symptoms PRO. Neurol Ther. 2023.
  32. Jackson K, Parthan A, Lauher-Charest M, Broderick L, Law N, Barnett C. Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient's Perspective: A Qualitative Study. Neurol Ther. 2023;12(1):107-128.
  33. Oosterhuis H. Clinical aspects. In: Myasthenia gravis: CRC press; 2019. p. 13-42.
  34. Rowin J. Approach to the patient with suspected myasthenia gravis or ALS: a clinician's guide. Continuum (Minneap Minn). 2009;15(1):13-34.
  35. Sanders DB, Wolfe GI, Benatar M, Evoli A, Gilhus NE, Illa I, et al. International consensus guidance for management of myasthenia gravis: Executive summary. Neurology. 2016;87(4):419-425.
  36. Myasthenia Gravis. https://www.ninds.nih.gov/health-information/disorders/myasthenia-gravis. Accessed 22 June.
  37. MGFA. MG Emergencies. https://myasthenia.org/MG-Community/-MG-Emergencies.
  38. Pascuzzi RM, Bodkin CL. Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome: New Developments in Diagnosis and Treatment. Neuropsychiatr Dis Treat. 2022;18:3001-3022.
  39. Narayanaswami P, Sanders DB, Wolfe G, Benatar M, Cea G, Evoli A, et al. International consensus guidance for management of myasthenia gravis: 2020 update. Neurology. 2021;96(3):114-122.
  40. Farrugia ME, Goodfellow JA. A practical approach to managing patients with myasthenia gravis-opinions and a review of the literature. Front Neurol. 2020;11:604.
  41. Nair SS, Jacob S. Novel immunotherapies for myasthenia gravis. Immunotargets Ther. 2023;12:25-45.
  42. Misra UK, Kalita J, Singh VK, Kumar S. A study of comorbidities in myasthenia gravis. Acta Neurol Belg. 2020;120(1):59-64.
  43. Nguyen MNL, van der Walt A, Fielding J, Clough M, White OB. Tracking eye movements for diagnosis in myasthenia gravis: A comprehensive review. J Neuroophthalmol. 2022;42(4):428-441.
  44. Dixon LB, Holoshitz Y, Nossel I. Treatment engagement of individuals experiencing mental illness: review and update. World Psychiatry. 2016;15(1):13-20.
  45. Alhaidar MK, Abumurad S, Soliven B, Rezania K. Current Treatment of Myasthenia Gravis. J Clin Med. 2022;11(6):1597.
  46. Farmakidis C, Pasnoor M, Dimachkie MM, Barohn RJ. Treatment of Myasthenia Gravis. Neurol Clin. 2018;36(2):311-337.
  47. Skeie GO, Apostolski S, Evoli A, Gilhus NE, Illa I, Harms L, et al. Guidelines for treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol. 2010;17(7):893-902.
  48. Benatar M, McDermott MP, Sanders DB, Wolfe GI, Barohn RJ, Nowak RJ, et al. Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME): A randomized, controlled trial. Muscle Nerve. 2016;53(3):363-369.
  49. Maggi L, Mantegazza R. Treatment of myasthenia gravis: focus on pyridostigmine. Clin Drug Investig. 2011;31(10):691-701.
  50. Tindall RS, Rollins JA, Phillips JT, Greenlee RG, Wells L, Belendiuk G. Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis. N Engl J Med. 1987;316(12):719-724.
  51. Hart IK, Sathasivam S, Sharshar T. Immunosuppressive agents for myasthenia gravis. Cochrane Database Syst Rev. 2007(4):CD005224.
  52. Drachman DB, Adams RN, Hu R, Jones RJ, Brodsky RA. Rebooting the immune system with high‐dose cyclophosphamide for treatment of refractory myasthenia gravis. Annals of the New York Academy of Sciences. 2008;1132(1):305-314.
  53. Skeie GO, Apostolski S, Evoli A, Gilhus NE, Hart IK, Harms L, et al. Guidelines for the treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol. 2006;13(7):691-699.
  54. Gajdos P, Chevret S, Toyka K. Plasma exchange for myasthenia gravis. Cochrane Database Syst Rev. 2002;2002(4):CD002275.
  55. Howard JF, Jr., Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, et al. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017;16(12):976-986.
  56. Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, Fujita KP, O'Brien FL, et al. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology. 2021;96(4):e610-e618.
  57. Soliris summary of product characteristics. 2022. Available at: https://www.ema.europa.eu/en/documents/product-information/soliris-epar-product-information_en.pdf. Accessed 31 January 2023.
  58. Soliris prescribing information. 2020. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125166s434lbl.pdf. Accessed 31 January 2023.
  59. Vu T, Meisel A, Mantegazza R, Annane D, Katsuno M, Aguzzi R, et al. Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. NEJM Evidence. 2022;1(5).
  60. Ultomiris (ravulizumab-cwvz) prescribing information. 2022. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761108s021lbl.pdf. Accessed 31 January 2023.
  61. Ravulizumab: Summary Of Product Characteristics. 2019. Available at: https://www.ema.europa.eu/en/documents/product-information/ultomiris-epar-product-information_en.pdf. Accessed 15 June 2023.
  62. Efgartigimod: Summary Of Product Characteristics. 2022. Available at: https://www.ema.europa.eu/en/documents/product-information/vyvgart-epar-product-information_en.pdf. Accessed 15 June 2023.
  63. Efgartigimod: Prescribing Information. 2021. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761195s000lbl.pdf. Accessed 15 June 2023.
  64. argenx Announces U.S. Food and Drug Administration Approval of VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) Injection for Subcutaneous Use in Generalized Myasthenia Gravis. https://www.globenewswire.com/en/news-release/2023/06/20/2691658/0/en/argenx-Announces-U-S-Food-and-Drug-Administration-Approval-of-VYVGART-Hytrulo-efgartigimod-alfa-and-hyaluronidase-qvfc-Injection-for-Subcutaneous-Use-in-Generalized-Myasthenia-Grav.html. Accessed 21 June.
  65. Kaminski H. Assessing the Growing Treatment Landscape for Myasthenia Gravis, Next Steps in Optimization: Henry Kaminski, MD. 2023.
  66. Menon D, Bril V. Pharmacotherapy of Generalized Myasthenia Gravis with Special Emphasis on Newer Biologicals. Drugs. 2022;82(8):865-887.
  67. Howard JF, Jr., Bril V, Vu T, Karam C, Peric S, Margania T, et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20(7):526-536.
  68. Saccà F, Barnett C, Vu T, Peric S, Phillips GA, Zhao S, et al. Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT). J Neurol. 2023;270(4):2096-2105.
  69. Bril V, Benatar M, Andersen H, Vissing J, Brock M, Greve B, et al. Efficacy and safety of rozanolixizumab in moderate to severe generalized myasthenia gravis: A phase 2 randomized control trial. Neurology. 2021;96(6):e853-e865.
  70. Rozanolixizumab: Prescribing Information. 2023. Available at: https://www.ucb-usa.com/RYSTIGGO-prescribing-information.pdf. Accessed 19 July 2023.
  71. UCB announces U.S. FDA approval of RYSTIGGO[®] (rozanolixizumab-noli) for the treatment of adults with generalized myasthenia gravis. https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-US-FDA-approval-of-RYSTIGGOR-rozanolixizumab-noli-for-the-treatment-of-adults-with-generalized-myasthenia-gravis. Accessed 19 July.
  72. Bril V, Drużdż A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394.
  73. Gable KL, Guptill JT. Antagonism of the neonatal Fc receptor as an emerging treatment for myasthenia gravis. Front Immunol. 2020;10:3052.
  74. Beer R, Blum S. Rozanolixizumab and Zilucoplan Phase 3 Generalised Myasthenia Gravis studies.
  75. Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo H-C, Marx A, et al. Randomized trial of thymectomy in myasthenia gravis. N Engl J Med. 2016;375(6):511-522.
  76. Hehir MK, Hobson-Webb LD, Benatar M, Barnett C, Silvestri NJ, Howard JF, Jr., et al. Rituximab as treatment for anti-MuSK myasthenia gravis: Multicenter blinded prospective review. Neurology. 2017;89(10):1069-1077.
  77. Tandan R, Hehir MK, 2nd, Waheed W, Howard DB. Rituximab treatment of myasthenia gravis: A systematic review. Muscle Nerve. 2017;56(2):185-196.
  78. Nowak RJ, Coffey CS, Goldstein JM, Dimachkie MM, Benatar M, Kissel JT, et al. Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study. Neurology. 2021;98(4):e376-389.
  79. Piehl F, Eriksson-Dufva A, Budzianowska A, Feresiadou A, Hansson W, Hietala MA, et al. Efficacy and Safety of Rituximab for New-Onset Generalized Myasthenia Gravis: The RINOMAX Randomized Clinical Trial. JAMA Neurol. 2022;79(11):1105-1112.
  80. Stergiou C, Lazaridis K, Zouvelou V, Tzartos J, Mantegazza R, Antozzi C, et al. Titin antibodies in "seronegative" myasthenia gravis--A new role for an old antigen. J Neuroimmunol. 2016;292:108-115.
  81. Cordts I, Bodart N, Hartmann K, Karagiorgou K, Tzartos JS, Mei L, et al. Screening for lipoprotein receptor-related protein 4-, agrin-, and titin-antibodies and exploring the autoimmune spectrum in myasthenia gravis. J Neurol. 2017;264(6):1193-1203.
  82. Nelke C, Schroeter CB, Stascheit F, Pawlitzki M, Regner-Nelke L, Huntemann N, et al. Eculizumab versus rituximab in generalised myasthenia gravis. J Neurol Neurosurg Psychiatry. 2022;93(5):548-554.
  83. Modrego P, Arribas J. Spontaneous resolution of a mediastinal mass in a woman with myasthenia gravis. Neurologia (Barcelona, Spain). 2016;33(8):556-557.
  84. Cutter G, Xin H, Aban I, Burns TM, Allman PH, Farzaneh-Far R, et al. Cross-sectional analysis of the Myasthenia Gravis Patient Registry: Disability and treatment. Muscle Nerve. 2019;60(6):707-715.
  85. Berrih-Aknin S, Palace J, Meisel A, Claeys KG, Muppidi S, Saccà F, et al. Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG). BMJ Open. 2023;13(5):e068104.
  86. Bozovic I, Ilic Zivojinovic J, Peric S, Kostic M, Ivanovic V, Lavrnic D, et al. Long-term outcome in patients with myasthenia gravis: one decade longitudinal study. J Neurol. 2022;269(4):2039-2045.
  87. Harris L, Graham S, MacLachlan S, Exuzides A, Jacob S. A retrospective longitudinal cohort study of the clinical burden in myasthenia gravis. BMC Neurol. 2022;22(1):172.
  88. Marlet IR, Andersen RK, Axelsen KH, Andersen LK, Vissing J, Witting N. Factors affecting the diagnostic delay of myasthenia gravis. J Neurol. 2024;272(1):29.
  89. Dragusin A, Grecu N, Ribigan AC, Badea RS, Terecoasa EO, Ene A, et al. Low Fluctuation of Symptoms May Delay Diagnosis of Myasthenia Gravis: A Case Series. Neurol Ther. 2022;11(1):481-487.
  90. Yoganathan K, Stevenson A, Tahir A, Sadler R, Radunovic A, Malek N. Bedside and laboratory diagnostic testing in myasthenia. Journal of Neurology. 2022;269(6):3372-3384.
  91. Gerischer L, Doksani P, Hoffmann S, Meisel A. New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making. BioDrugs. 2025;39(2):185-213.
  92. Funke M, Eveslage M, Zschüntzsch J, Hagenacker T, Ruck T, Schubert C, et al. Fatigue and associated factors in myasthenia gravis: a nationwide registry study. Journal of Neurology. 2024;271(8):5665-5670.
  93. Alekseeva TM, Gavrilov YV, Kreis OA, Valko PO, Weber KP, Valko Y. Fatigue in patients with myasthenia gravis. Journal of Neurology. 2018;265(10):2312-2321.
  94. Jackson K, Parthan A, Lauher-Charest M, Broderick L, Law N, Barnett C. Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient’s Perspective: A Qualitative Study. Neurology and Therapy. 2023;12(1):107-128.
  95. Zhou J, Nilius S, Pilipczuk O, Scowcroft A, Tarancón T, Tennigkeit F, et al. Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States. Neurology. 2025;105(1):e213736.
  96. Reza Ashraghi M, Myasthenia gravis and the elderly. Advances in Clinical Neuroscience and Rehabilation. 2024.
  97. Bauchner H, Rivara FP. The Status of Pediatric Randomized Clinical Trials. The Journal of Pediatrics. 2024;266.
  98. Chanson JB, Bouhour F, Aubé-Nathier AC, Mallaret M, Vial C, Hacquard A, et al. Myasthenia gravis treatment in the elderly presents with a significant iatrogenic risk: a multicentric retrospective study. Journal of Neurology. 2023;270(12):5819-5826.
  99. Lai R. Treating Older PatientsWith Late-Onset Myasthenia Gravis. https://www.rarediseaseadvisor.com/insights/treating-elderly-patients-myasthenia-gravis/.
  100. Sikorski P, Li Y, Cheema M, Wolfe GI, Kusner LL, Aban I, et al. Serum metabolomics of treatment response in myasthenia gravis. PLoS One. 2023;18(10):e0287654.
  101. UCB announces U.S. FDA acceptance of new drug application and EMA MAA validation for zilucoplan for the treatment of generalized myasthenia gravis in adult patients. https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-US-FDA-acceptance-of-new-drug-application-and-EMA-MAA-validation-for-zilucoplan-for-the-treatment-of-generalized-myasthenia-gravis-in-adult-patients. Accessed 20 June.
  102. Howard JF, Jr., Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, et al. Clinical effects of the self-administered subcutaneous complement inhibitor zilucoplan in patients with moderate to severe generalized myasthenia gravis: Results of a phase 2 randomized, double-blind, placebo-controlled, multicenter clinical trial. JAMA Neurol. 2020;77(5):582-592.
  103. Howard JF, Jr., Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023;22(5):395-406.
  104. Nowak RJ, Breiner A, Bril V, Allen JA, Khan S, Levine T, et al. Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open-label extension. Ann Clin Transl Neurol. 2024;11(1):194-206.
  105. Immunovant. Immunovant Announces Positive Results for Batoclimab Myasthenia Gravis (MG) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Studies. https://www.immunovant.com/investors/news-events/press-releases/detail/71/immunovant-announces-positive-results-for-batoclimab.
  106. Barratt J, Liew A, Yeo SC, Fernström A, Barbour SJ, Sperati CJ, et al. Phase 2 Trial of Cemdisiran in Adult Patients with IgA Nephropathy: A Randomized Controlled Trial. Clin J Am Soc Nephrol. 2024;19(4):452-462.
  107. Regeneron. Regeneron Announces Positive Results from Phase 3 Trial in Generalized Myasthenia Gravis. https://investor.regeneron.com/news-releases/news-release-details/regeneron-announces-positive-results-phase-3-trial-generalized.
  108. Studna A. Phase III NIMBLE Trial Demonstrates Cemdisiran Efficacy in Generalized Myasthenia Gravis. Applied Clinical Trials. 2025.