ORPHA:589
Myasthenia gravis
Also known as: Acquired myasthenia · Autoimmune myasthenia gravis
Publications
47,233
98.7th percentile
Trials
224
Interventional, condition-specific
Researchers
1,079
Distinct authors in sample
Gene link
—
Readiness
4/6
Stages with a signal
Clinical definition (Orphanet)
A rare autoimmune disorder of the neuromuscular junction characterized by fatigable weakness of voluntary muscles.
How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0009688
- MeSH:D009157
- OMIM:254200
- UMLS:C0026896
- NCIT:C60989
Additional Mondo synonyms (3)
acquired myasthenia · autoimmune myasthenia gravis · myasthenia gravis
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
4/6 stages with a signal
An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
47,233 matched papers (22,158 in last 10 years) Source
- Phenotype characterisedPresent
73 HPO annotations (e.g. Psychosis; Acrocyanosis; Seizure) Source
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationPresent
11 FDA · 9 EMA designations (11 FDA orphan-indication approvals) — e.g. satralizumab-mwge Source
- Interventional trialPresent
224 matched on ClinicalTrials.gov (92 recruiting in sample)
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
73
Associated phenotypes · MONDO:0009688
- Psychosis
- Acrocyanosis
- Seizure
- Hemolytic anemia
- Hepatitis
Showing 5 of 73 — open Monarch for the full list.
Animal models (Monarch / Alliance)
None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.
Monarch fetch 2026-07-29
Therapies
Designations, candidates, and chemicals
FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.
Orphan designation (FDA · EMA)
23
Designations · 11 with FDA orphan-indication approval
- FDA satralizumab-mwgeMyasthenia Gravis · 2021-02-23 · Not FDA Approved for Orphan Indication
- FDA nipocalimabMyasthenia Gravis · 2021-02-11 · Not FDA Approved for Orphan Indication
- FDA ZilucoplanMyasthenia Gravis · 2019-08-26 · Not FDA Approved for Orphan Indication
- FDA rozanolixizumabMyasthenia Gravis · 2019-02-01 · Not FDA Approved for Orphan Indication
- FDA efgartigimodMyasthenia Gravis · 2017-09-20 · Not FDA Approved for Orphan Indication
- FDA immune globulin (human)Myasthenia Gravis · 2016-12-12 · Not FDA Approved for Orphan Indication
- FDA amifampridine phosphateMyasthenia Gravis · 2016-08-31 · Not FDA Approved for Orphan Indication
- FDA MethotrexateMyasthenia Gravis · 2014-08-28 · Not FDA Approved for Orphan Indication
Sources: FDA OOPD · EMA orphan designations
Open Targets candidates
78
Drugs / clinical candidates · MONDO_0009688
- AZATHIOPRINE·phase 3
- BATOCLIMAB·phase 3
- BQ 788·phase 3
- CEMDISIRAN·phase 3
- CLADRIBINE·phase 3
- ECULIZUMAB·phase 3
- GEFURULIMAB·phase 3
- HUMAN IMMUNOGLOBULIN G·phase 3
- INEBILIZUMAB·phase 3
- IPTACOPAN·phase 3
- LEFLUNOMIDE·phase 3
- MYCOPHENOLATE MOFETIL·phase 3
- POZELIMAB·phase 3
- PREDNISONE·phase 3
- RAVULIZUMAB·phase 3
CTD chemicals (MyDisease.info)
54 associated chemicals · 118 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.
- Ambenonium Chloride · therapeutic
- Atropine · therapeutic
- Azathioprine · therapeutic
- Calcium Gluconate · therapeutic
- Cholinesterase Inhibitors · therapeutic
- Clonazepam · therapeutic
- Cyclosporine · therapeutic
- Edrophonium · therapeutic
- Glucocorticoids · therapeutic
- Hydroxocobalamin · therapeutic
- Mycophenolic Acid · therapeutic
- Neostigmine · therapeutic
Pathways: Platinum drug resistance; MAPK signaling pathway; Cytokine-cytokine receptor interaction; p53 signaling pathway; Endocytosis; Phagosome; Apoptosis; Cell adhesion molecules (CAMs)
Literature
Is anyone studying this?
47,233
47,233 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
47,233 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).
22,158 in the last 10 years · high confidence · 98.7th percentile (publications denominator)
Phrase hits: 47,233 · MeSH hits: 0
Who's working on it?
1,079
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01Li Y14 papers · 2026
Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.
Papers in Europe PMC - 02Zhang Y12 papers · 2026
Guangzhou University of Chinese Medicine, Guangzhou, China.
Papers in Europe PMC - 03Wang J7 papers · 2026
Department of Encephalopathy, The Affiliated Hospital to Changchun University of Chinese Medicine, Changchun, Jilin, China.
Papers in Europe PMC - 04Zhang H7 papers · 2026
Eye Center of Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.
Papers in Europe PMC - 05Zhang X7 papers · 2026
Department of Neurology, The 940 Hospital of Joint Logistic Support Forces of PLA, Lanzhou, China. Electronic address: zhangxiaoyan060110@163.com.
Papers in Europe PMC - 06Meisel A6 papers · 2026
Department of Neurology with Experimental Neurology, Neuroscience Clinical Resarch Center, Charite Universitatsmedizin Berlin, Berlin, Germany.
Papers in Europe PMC - 07Zhou Y6 papers · 2026
Department of Thoracic Surgery, The People's Hospital of Guangxi Zhuang Autonomous Region, Guangxi Academy of Medical Sciences, Nanning, China.
Papers in Europe PMC - 08Vissing J5 papers · 2026
Copenhagen Neuromuscular Center, Rigshospitalet, Inge Lehmanns Vej 8, Copenhagen, Denmark.
Papers in Europe PMC - 09Zhang Z5 papers · 2026
Department of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, China.
Papers in Europe PMC - 10Chen H4 papers · 2026
Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing, China.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
224
interventional trials for this specific condition
224 interventional trials matched this specific condition name; 92 currently recruiting in our sample.
Data as of 11 September 2026
224 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 99.4th percentile).
high confidence · 99.4th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
224 interventional trials matched after quoted-phrase search and title/condition post-filter.
- NCT06371040·RECRUITING·Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT05868837·RECRUITING·Rituximab EfFicacy IN MyasthEnia Gravis (REFINE)
Not reviewed·Conditions: Myasthenia Gravis, Generalized·Matched via name phrase
- NCT06414954·RECRUITING·Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis · Myasthenia Gravis, MuSK·Matched via name phrase
- NCT06193889·RECRUITING·KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis · Generalized Myasthenia Gravis·Matched via name phrase
- NCT07215949·RECRUITING·Zilucoplan for Severe gMG Exacerbations
Not reviewed·Conditions: Generalized Myasthenia Gravis (gMG)·Matched via name phrase
- NCT06688435·RECRUITING·A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells in Patients With Refractory Generalized Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT06939166·RECRUITING·Universal Chimeric Antigen Receptor T-Cell (UCAR T-cell) Therapy Targeting CD19/B Cell Maturation Antigen (CD19/BCMA) in Patients With r/r Neurological Autoimmune Diseases
Not reviewed·Conditions: Neuromyelitis Optica Spectrum Disorders · Myasthenia Gravis · Multiple Sclerosis · Chronic Inflammatory Demyelinating Polyradiculoneuropathy·Matched via name phrase
- NCT07250750·RECRUITING·A Phase 1b/2 Study of IM-101 in Adult Participants With Generalized Myasthenia Gravis and Ocular Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT06836973·NOT YET RECRUITING·Blinatumomab for Treatment of Refractory Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT06447597·RECRUITING·A Clinical Study of B007 in the Treatment of Generalized Myasthenia Gravis.
Not reviewed·Conditions: Generalized Myasthenia Gravis·Matched via name phrase
- NCT06799247·RECRUITING·Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis
Not reviewed·Conditions: Myasthaenia Gravis·Matched via name phrase
- NCT07221838·RECRUITING·A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
Not reviewed·Conditions: Generalized Myasthenia Gravis · gMG·Matched via name phrase
- NCT06759948·NOT YET RECRUITING·An Early Exploratory Clinical Study of GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis
Not reviewed·Conditions: Generalized Myasthenia Gravis (gMG)·Matched via name phrase
- NCT04833894·RECRUITING·Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Not reviewed·Conditions: Generalized Myasthenia Gravis·Matched via name phrase
- NCT04561557·RECRUITING·Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
Not reviewed·Conditions: Autoimmune Diseases · Autoimmune Diseases of the Nervous System · Neuromyelitis Optica Spectrum Disorder · Myasthenia Gravis·Matched via name phrase
Observational and natural-history studies
103 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.
Recruiting or not-yet-recruiting
- NCT06866652·RECRUITING·Evaluating Dyspnea in Autoimmune Myasthenia Gravis "Why am I Short of Breath?"
Not reviewed·Conditions: Myasthenia Gravis · Dyspnea·Matched via name phrase
- NCT07294170·RECRUITING·ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis · MG · gMG · Generalized Myasthenia Gravis·Matched via name phrase
- NCT07226830·NOT YET RECRUITING·Quantification of Change in MG Disease Activity in Individuals With Generalized Myasthenia Gravis (gMG) After Administration of VYVGART® or VYVGART Hytrulo® Using BioDigit MG
Not reviewed·Conditions: Myasthenia Gravis Generalized·Matched via name phrase
- NCT07247279·RECRUITING·Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia
Not reviewed·Conditions: Rare Diseases · Generalized Myasthenia Gravis (gMG)·Matched via name phrase
- NCT06312644·RECRUITING·Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Not reviewed·Conditions: Ultomiris-exposed Pregnant/ Postpartum · Pregnancy · Paroxysmal Nocturnal Hemoglobinuria (PNH) · Atypical Hemolytic Uremic Syndrome (aHUS)·Matched via name phrase
- NCT06064461·RECRUITING·Vitaccess Real MG Registry
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT06866483·RECRUITING·Validation and Scaling of Screening Program for Undiagnosed Myasthenia Gravis-Social Media Campaign Paired With a Self-moderated Assessment
Not reviewed·Conditions: Myasthenia Gravis · Neuromuscular Disease · Neuromuscular Manifestations·Matched via name phrase
- NCT04837625·RECRUITING·Study of Myasthenic Crisis in China
Not reviewed·Conditions: Myasthenia Gravis · Myasthenic Crisis·Matched via name phrase
- NCT07677852·NOT YET RECRUITING·Sexual and Urinary Dysfunctions in Generalized Myasthenia
Not reviewed·Conditions: Myasthenia Gravis (MG)·Matched via name phrase
- NCT06815133·RECRUITING·Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT04202341·RECRUITING·Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
Not reviewed·Conditions: Generalized Myasthenia Gravis·Matched via name phrase
- NCT07717281·RECRUITING·PANORAMA: Neuromuscular Organoids for Refractory AChR+ Myasthenia Gravis
Not reviewed·Conditions: Myasthenia Gravis Generalized·Matched via name phrase
- NCT06006832·RECRUITING·A Prospective Cohort Study of Myasthenia Gravis in China
Not reviewed·Conditions: Myasthenia Gravis·Matched via name phrase
- NCT07570589·RECRUITING·Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)
Not reviewed·Conditions: Generalized Myasthenia Gravis·Matched via name phrase
- NCT05214612·RECRUITING·Predictors and Prognostic Factors of Myasthenia Gravis Outcome
Not reviewed·Conditions: Myasthenia Gravis · Autoimmune Diseases of the Nervous System · Neuromuscular Junction Diseases · Thymoma·Matched via name phrase
General rare disease registries you may be eligible for
These studies enroll across many rare conditions. They are not counted as evidence that anyone is studying this specific disease.
- NCT01793168·RECRUITING·Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Not reviewed·Conditions: Rare Disorders · Undiagnosed Disorders · Disorders of Unknown Prevalence · Cornelia De Lange Syndrome
Other registries (secondary)
Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.
raw 101 · after dedupe 97 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 97 · dropped 0 · fetched 2026-07-29
Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri
No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).
Uncertain / not reviewed (97)
- isrctn·ISRCTN14011923·Recruiting·Higher protein intake after intensive care
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN42000162·Recruiting·Using ultrasound to improve antibiotic treatment in infected burns: a feasibility study
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN22102770·Recruiting·A study to test the safety and effects of a New Drug (LAE103) in healthy people who are overweight or obese, and in healthy postmenopausal women. The study also looks at how LAE103 works when taken alone or together with another drug (LAE102).
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN14932955·No longer recruiting·A split-face study comparing intradermal botulinum toxin A with and without hyaluronic acid biorevitalization for facial rejuvenation
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN26415952·Recruiting·A Study to evaluate novel KarX and KarT Prototypes versus the KarXT and KarX-EC reference following single doses, and to explore the effect of food after multiple doses of selected prototypes in healthy adult participants
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN82186642·No longer recruiting·Research on analgesia in mechanically ventilated patients following abdominal surgery
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN11134984·Recruiting·CLArithromycin for post-Stroke Pneumonia
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN58670024·No longer recruiting·The effect of adjunctive triple antibiotic paste in deep periodontal pocket management: a comprehensive investigation of clinical, immunological and microbiological outcomes through a randomised controlled trial
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN30709760·Recruiting·A randomised trial comparing two current ventilation treatments in the Intensive Care Unit: The UK NAVA Trial
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN13274002·Recruiting·Feasibility and effects of using ketamine sedation for patients on the Intensive Care Unit.
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN83274361·No longer recruiting·A phase Ib study to investigate safety and tolerability of ARGX-119 in adult participants with DOK7 congenital myasthenic syndrome (CMS)
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN18175685·No longer recruiting·Stratification of clinically vulnerable people for COVID-19 risk using antibody testing
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN17158033·Recruiting·Does setting the breathing machine (ventilator) to deliver breaths using a method called airway pressure release ventilation help patients with diseased lungs to heal faster and spend less time on a ventilator?
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN13629437·No longer recruiting·Plasma exchange therapy with the crossflow membrane filtration method for post-COVID-19 conditions
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN36536677·Recruiting·A trial of medications in comparison to dummy tablets for the treatment of drooling caused by clozapine.
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN10681798·No longer recruiting·A study to investigate the safety, tolerability, disposition in the body, effects of RO7308480 on the body, and its changes in midazolam disposition in the body following oral administration in healthy participants
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN68072469·No longer recruiting·Using minocycline to help to identify carotid artery disease that may cause future strokes
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN31763161·Stopped·A study to test the safety and immune response in adult humans of a new vaccine against four different types of Shigella bacteria
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN60460377·No longer recruiting·The effect of a magnesium supplement on arterial calcification and arterial stiffness in individuals with type 2 diabetes
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN18340272·No longer recruiting·An observational longitudinal study of congenital myasthenic syndromes
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN10010669·No longer recruiting·A study to assess the effect of tiragolumab in presence of atezolizumab and bevacizumab in participants detected with lung cancer
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN83474167·No longer recruiting·Phase II open-label trial of atezolizumab in patients with urinary tract squamous cell carcinoma
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN72886945·Recruiting·Effect of two different ventilator weaning modes on patients with difficulty and delayed weaning
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN79243381·No longer recruiting·Investigating a vaccine against plague in Uganda (PlaVac Uganda)
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN91879618·No longer recruiting·The effects on the body and processing by the body of biperiden injected into the blood in elderly subjects
skipped — LLM skipped (--skip-llm)
Where to find support
Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.
Orphanet entry for Myasthenia gravis — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Myasthenia gravis" OR "Acquired myasthenia" OR "Autoimmune myasthenia gravis"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Myasthenia gravis" OR "Acquired myasthenia" OR "Autoimmune myasthenia gravis"
Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).
Study-type breakdown: 224 interventional · 103 observational · 0 expanded access. Only interventional studies enter the trial headline.
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Confidence reasoning
- Preferred label is multi-word and distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus
Ingested 2026-07-26T14:28:37.150Z
