RARE DISEASERESEARCH ATLAS

ORPHA:589

Myasthenia gravis

high confidenceDisorder

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.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

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.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    47,233 matched papers (22,158 in last 10 years) Source

  3. Phenotype characterisedPresent

    73 HPO annotations (e.g. Psychosis; Acrocyanosis; Seizure) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    11 FDA · 9 EMA designations (11 FDA orphan-indication approvals) — e.g. satralizumab-mwge Source

  6. 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

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)

MyDisease.info · MONDO:0009688

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

Open Europe PMC search

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.

  1. 01
    Li Y14 papers · 2026

    Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.

    Papers in Europe PMC
  2. 02
    Zhang Y12 papers · 2026

    Guangzhou University of Chinese Medicine, Guangzhou, China.

    Papers in Europe PMC
  3. 03
    Wang J7 papers · 2026

    Department of Encephalopathy, The Affiliated Hospital to Changchun University of Chinese Medicine, Changchun, Jilin, China.

    Papers in Europe PMC
  4. 04
    Zhang H7 papers · 2026

    Eye Center of Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.

    Papers in Europe PMC
  5. 05
    Zhang 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
  6. 06
    Meisel A6 papers · 2026

    Department of Neurology with Experimental Neurology, Neuroscience Clinical Resarch Center, Charite Universitatsmedizin Berlin, Berlin, Germany.

    Papers in Europe PMC
  7. 07
    Zhou 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
  8. 08
    Vissing J5 papers · 2026

    Copenhagen Neuromuscular Center, Rigshospitalet, Inge Lehmanns Vej 8, Copenhagen, Denmark.

    Papers in Europe PMC
  9. 09
    Zhang Z5 papers · 2026

    Department of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, China.

    Papers in Europe PMC
  10. 10
    Chen 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.

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

Open the complete matched search on ClinicalTrials.gov

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.

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)

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 2026-07-26

This 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"

Run this search on Europe PMC

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