Biomarker & Intervention Discovery

Myasthenia Gravis (MG)

This page lists 10 druggable gene targets, 48 approved or trial-tested drug candidates and 15 recent clinical trials for Myasthenia Gravis (MG), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

NIH funding$7MFY2025 · RCDC estimate
Funding levelModerate
Spontaneous remissionSpontaneous complete stable remission (CSR) in 10-20% of patients over 10 years; generalised MG rarely resolves…
Best-intervention remissionThymectomy in non-thymomatous MG at age <65: 50% more likely to achieve CSR at 3 years vs medical therapy (MGTX…
Gap sizeLarge — new complement inhibitors and FcRn antagonists represent genuine advances but cost and access are barriers…
Primary barrieraccess
Full remission profile →
Research Tracker Chronic Disease Interventions Myasthenia Gravis (MG)

Remission & chronicity

Spontaneous remission
Spontaneous complete stable remission (CSR) in 10-20% of patients over 10 years; generalised MG rarely resolves without treatment; ocular MG may remain limited in 50-60%
Best-intervention remission
Thymectomy in non-thymomatous MG at age <65: 50% more likely to achieve CSR at 3 years vs medical therapy (MGTX trial); eculizumab (ravulizumab): 40% reduction in exacerbations in AChR+ MG; efgartigimod alfa (Vyvgart): 67.7% MG-ADL responders (ADAPT trial); zilucoplan: 53% MG-ADL response (RAISE trial)
Gap size
Large — new complement inhibitors and FcRn antagonists represent genuine advances but cost and access are barriers; thymectomy underused in appropriate candidates
Primary barrier
access
Thymectomy (MGTX trial proven benefit) is delayed or not offered in many patients with non-thymomatous MG; eculizumab ($700K/year) dramatically limits access; efgartigimod and zilucoplan more accessible but still $200K+/year; corticosteroids cause significant morbidity in MG patients managed long-term

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

ACHE 233C5 23CD19 40CHRNA1 63CHRNE 57FCGRT 120IL6R 13LRP4 0MS4A1 28MUSK 37

ACHE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
DEMECARIUM BROMIDE ? unclear Mechanistic DGIdb
LADOSTIGIL ? unclear Mechanistic DGIdb
ISOFLUROPHATE ↓ inhibits Mechanistic DGIdb
GALANTAMINE ↓ inhibits Mechanistic DGIdb
DONEPEZIL ↓ inhibits Mechanistic 2.33 nM (IC50) DGIdb
TACRINE ↓ inhibits Mechanistic 218.78 nM (IC50) DGIdb
COMPOUND 2F [JIANG ET AL., 2018] ↓ inhibits Mechanistic DGIdb
EDROPHONIUM CATION ↓ inhibits Mechanistic DGIdb
SKIMMIANINE ? unclear Mechanistic DGIdb
ACOTIAMIDE ? unclear Mechanistic DGIdb
MIMOPEZIL ? unclear Mechanistic DGIdb
PROPANIDID ↓ inhibits Mechanistic DGIdb
DIANHYDROGALACTITOL ? unclear Mechanistic DGIdb
PRALIDOXIME CHLORIDE ↑ activates Mechanistic DGIdb
AMBENONIUM ? unclear Mechanistic DGIdb
6-AMINONICOTINAMIDE ? unclear Mechanistic DGIdb
SURONACRINE MALEATE ? unclear Mechanistic DGIdb
PHENSERINE ? unclear Mechanistic DGIdb
METHADONE HYDROCHLORIDE ? unclear Mechanistic DGIdb
LATREPIRDINE ? unclear Mechanistic DGIdb
PHYSOSTIGMINE ↓ inhibits Mechanistic 30.0 nM (IC50) DGIdb
MAGNESIUM CHLORIDE ANHYDROUS ? unclear Mechanistic DGIdb
METHANESULFONYL FLUORIDE ? unclear Mechanistic DGIdb
DONECOPRIDE ↓ inhibits Mechanistic DGIdb
PYRIDOSTIGMINE BROMIDE ↓ inhibits Mechanistic DGIdb
EPTASTIGMINE ? unclear Mechanistic 290.0 nM (IC50) DGIdb
BETAMETHASONE ? unclear Mechanistic DGIdb
MINAPRINE ↓ inhibits Mechanistic DGIdb
HUPERZINE A ↓ inhibits Mechanistic 5.0 nM (IC50) DGIdb
NEOSTIGMINE ↓ inhibits Mechanistic DGIdb
MALATHION ? unclear Mechanistic DGIdb
DONEPEZIL HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
ACHE REACTIVATOR 2 ? unclear Mechanistic DGIdb
BW284C51 ↓ inhibits Mechanistic DGIdb
EDROPHONIUM CHLORIDE ↓ inhibits Mechanistic DGIdb
ITOPRIDE ? unclear Mechanistic DGIdb
BUNTANETAP ? unclear Mechanistic DGIdb
8-AZAGUANINE ? unclear Mechanistic DGIdb
HEXAFLUORENIUM BROMIDE ↓ inhibits Mechanistic DGIdb
RIVASTIGMINE TARTRATE ↓ inhibits Mechanistic DGIdb

Showing top 40 of 233 agents ranked by evidence tier.

C5

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ZILUCOPLAN ↓ inhibits Mechanistic DGIdb
ECULIZUMAB ↓ inhibits Mechanistic DGIdb
TESIDOLUMAB ~ modulates Mechanistic DGIdb
OLENDALIZUMAB ↓ inhibits Mechanistic DGIdb
POZELIMAB ↓ inhibits Mechanistic DGIdb
CROVALIMAB ↓ inhibits Mechanistic DGIdb
AVACINCAPTAD PEGOL ↓ inhibits Mechanistic DGIdb
RAVULIZUMAB ↓ inhibits Mechanistic DGIdb
ZILUCOPLAN SODIUM ~ modulates Mechanistic DGIdb
VILOBELIMAB ↓ inhibits Mechanistic DGIdb
PEXELIZUMAB ↓ inhibits Mechanistic DGIdb
NOMACOPAN ↓ inhibits Mechanistic DGIdb
AVACINCAPTAD PEGOL SODIUM ? unclear Mechanistic Open Targets
CARPROFEN ↓ inhibits Mechanistic 580.0 nM (Kd) ChEMBL
OXAPROZIN ↓ inhibits Mechanistic 640.0 nM (Kd) ChEMBL
SULINDAC ↓ inhibits Mechanistic 570.0 nM (Kd) ChEMBL
RALOXIFENE ↓ inhibits Mechanistic 710.0 nM (Kd) ChEMBL
CHEMBL2204196 ↓ inhibits Mechanistic 440.0 nM (IC50) ChEMBL
CHEMBL2203296 ↓ inhibits Mechanistic 210.0 nM (IC50) ChEMBL
CHEMBL4756677 ↓ inhibits Mechanistic 620.0 nM (IC50) ChEMBL
CHEMBL5594087 ↓ inhibits Mechanistic 940.0 nM (Kd) ChEMBL
CHEMBL5594178 ↓ inhibits Mechanistic 700.0 nM (Kd) ChEMBL
CEMDISIRAN ? unclear Clinical Open Targets

CD19

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
AUTOLOGOUS ANTI-CD19 CAR T-CELLS IM19 ? unclear Mechanistic DGIdb
BLINATUMOMAB ? unclear Mechanistic DGIdb
LONCASTUXIMAB TESIRINE ~ modulates Mechanistic DGIdb
AUTOLOGOUS CD19CAR-CD28-CD137/CD27/CD3ZETA-ICASP9-EXPRESSING T-LYMPHOCYTES ? unclear Mechanistic DGIdb
TISAGENLECLEUCEL ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD19 CHIMERIC ANTIGEN RECEPTOR T-CELLS C-CAR011 ? unclear Mechanistic DGIdb
VADACABTAGENE LERALEUCEL ? unclear Mechanistic DGIdb
LISOCABTAGENE MARALEUCEL ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD19CAR-4-1BB-CD3ZETA-EGFRT-EXPRESSING T LYMPHOCYTES ? unclear Mechanistic DGIdb
MDX-1342 ~ modulates Mechanistic DGIdb
AXICABTAGENE CILOLEUCEL ? unclear Mechanistic DGIdb
ANTI-CD19 CAR T CELLS PREPARATION ? unclear Mechanistic DGIdb
INEBILIZUMAB ? unclear Mechanistic DGIdb
ALLOGENEIC ANTI-CD19-CAR-CD28-IL-15-EXPRESSING NATURAL KILLER T-CELLS ? unclear Mechanistic DGIdb
AZERCABTAGENE ZAPRELEUCEL ? unclear Mechanistic DGIdb
TAFASITAMAB ? unclear Mechanistic DGIdb
DENINTUZUMAB MAFODOTIN ~ modulates Mechanistic DGIdb
COLTUXIMAB RAVTANSINE ? unclear Mechanistic DGIdb
OBEXELIMAB ↓ inhibits Mechanistic DGIdb
AMG-729 ~ modulates Mechanistic DGIdb
CD19CAR-CD3ZETA-4-1BB-EXPRESSING ALLOGENEIC T-LYMPHOCYTE CELLS ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD19 CAR-T CELLS TBI-1501 ? unclear Mechanistic DGIdb
AUTOLOGOUS CD19 CAR-EXPRESSING CD4+/CD8+ T-CELLS MB-CART19.1 ? unclear Mechanistic DGIdb
AFM-11 ? unclear Mechanistic DGIdb
BREXUCABTAGENE AUTOLEUCEL ~ modulates Mechanistic DGIdb
TAPLITUMOMAB PAPTOX ~ modulates Mechanistic DGIdb
ANTI-CD19 CAR T-CELLS XLCART001 ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD19 CAR-EXPRESSING T-LYMPHOCYTES CLIC-1901 ? unclear Mechanistic DGIdb
DENINTUZUMAB ~ modulates Mechanistic DGIdb
AUTO3 ? unclear Mechanistic DGIdb
DEGLYCOSYLATED RICIN A CHAIN-CONJUGATED ANTI-CD19/ANTI-CD22 IMMUNOTOXINS ? unclear Mechanistic DGIdb
DUVORTUXIZUMAB ? unclear Mechanistic DGIdb
ANTI-CD19 CAR-T CELLS ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD19 CHIMERIC ANTIGEN RECEPTOR T-CELLS SJCAR19 ? unclear Mechanistic DGIdb
ONCOLYSIN B ~ modulates Mechanistic DGIdb
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic DGIdb
CD19-TARGETED CAR T2 CELLS ? unclear Mechanistic DGIdb
OBECABTAGENE AUTOLEUCEL ~ modulates Mechanistic DGIdb
JCAR014 ? unclear Mechanistic DGIdb
RELMACABTAGENE AUTOLEUCEL ? unclear Mechanistic DGIdb

CHRNA1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
GALLAMINE TRIETHIODIDE ↓ inhibits Mechanistic DGIdb
ATRACURIUM BESYLATE ↓ inhibits Mechanistic DGIdb
EFGARTIGIMOD ALFA ? unclear Mechanistic DGIdb
DECAMETHONIUM BROMIDE ↑ activates Mechanistic DGIdb
MIVACURIUM CHLORIDE ↓ inhibits Mechanistic DGIdb
ZILUCOPLAN ? unclear Mechanistic DGIdb
ECULIZUMAB ? unclear Mechanistic DGIdb
NIPOCALIMAB ? unclear Mechanistic DGIdb
PANCURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
METOCURINE IODIDE ↓ inhibits Mechanistic DGIdb
DOXACURIUM ↓ inhibits Mechanistic DGIdb
RAVULIZUMAB ? unclear Mechanistic DGIdb
MECAMYLAMINE ↓ inhibits Mechanistic DGIdb
TUBOCURARINE CHLORIDE ↓ inhibits Mechanistic DGIdb
[3H]&ALPHA;-BUNGAROTOXIN ↓ inhibits Mechanistic DGIdb
SUCCINYLCHOLINE ↑ activates Mechanistic DGIdb
&ALPHA;-CONOTOXIN MI ↓ inhibits Mechanistic DGIdb
&ALPHA;-CONOTOXIN GI ↓ inhibits Mechanistic DGIdb
WAGLERIN-1 ↓ inhibits Mechanistic DGIdb
DECAMETHONIUM ? unclear Mechanistic DGIdb
PANCURONIUM ↓ inhibits Mechanistic DGIdb
ROZANOLIXIZUMAB ? unclear Mechanistic DGIdb
VECURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
RAPACURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
&ALPHA;-BUNGAROTOXIN ↓ inhibits Mechanistic DGIdb
PIPECURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
CISATRACURIUM BESYLATE ↓ inhibits Mechanistic DGIdb
ROCURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
SUCCINYLCHOLINE CHLORIDE ↑ activates Mechanistic DGIdb
ATRACURIUM ↓ inhibits Mechanistic DGIdb
[125I]&ALPHA;-BUNGAROTOXIN ↓ inhibits Mechanistic DGIdb
PIPECURONIUM ? unclear Mechanistic Open Targets
TUBOCURARINE ? unclear Mechanistic Open Targets
CHEMBL84149 ↑ activates Mechanistic 34000.0 nM (EC50) ChEMBL
CHEMBL1210703 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL1210515 ↓ inhibits Mechanistic 794.33 nM (IC50) ChEMBL
CHEMBL1823380 ↓ inhibits Mechanistic 4200.0 nM (IC50) ChEMBL
CHEMBL1823381 ↓ inhibits Mechanistic 5500.0 nM (IC50) ChEMBL
CHEMBL1823382 ↓ inhibits Mechanistic 7800.0 nM (IC50) ChEMBL
CHEMBL2022495 ↓ inhibits Mechanistic 3981.07 nM (IC50) ChEMBL

Showing top 40 of 63 agents ranked by evidence tier.

CHRNE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
GALLAMINE TRIETHIODIDE ↓ inhibits Mechanistic DGIdb
ZILUCOPLAN ? unclear Mechanistic DGIdb
PIPECURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
CISATRACURIUM BESYLATE ↓ inhibits Mechanistic DGIdb
PANCURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
EFGARTIGIMOD ALFA ? unclear Mechanistic DGIdb
DOXACURIUM ↓ inhibits Mechanistic DGIdb
RAPACURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
ECULIZUMAB ? unclear Mechanistic DGIdb
DECAMETHONIUM BROMIDE ↑ activates Mechanistic DGIdb
RAVULIZUMAB ? unclear Mechanistic DGIdb
ATRACURIUM BESYLATE ↓ inhibits Mechanistic DGIdb
VECURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
NIPOCALIMAB ? unclear Mechanistic DGIdb
TUBOCURARINE CHLORIDE ↓ inhibits Mechanistic DGIdb
ROZANOLIXIZUMAB ? unclear Mechanistic DGIdb
METOCURINE IODIDE ↓ inhibits Mechanistic DGIdb
SUCCINYLCHOLINE CHLORIDE ↑ activates Mechanistic DGIdb
MIVACURIUM CHLORIDE ↓ inhibits Mechanistic DGIdb
ROCURONIUM BROMIDE ↓ inhibits Mechanistic DGIdb
PIPECURONIUM ? unclear Mechanistic Open Targets
PANCURONIUM ? unclear Mechanistic Open Targets
TUBOCURARINE ? unclear Mechanistic Open Targets
DECAMETHONIUM ? unclear Mechanistic Open Targets
TACRINE ↓ inhibits Mechanistic 44.0 nM (IC50) ChEMBL
CHEMBL224906 ↓ inhibits Mechanistic 125.0 nM (IC50) ChEMBL
CHEMBL4228909 ↓ inhibits Mechanistic 50.0 nM (IC50) ChEMBL
CHEMBL4471876 ↓ inhibits Mechanistic 27.0 nM (IC50) ChEMBL
CHEMBL4435544 ↓ inhibits Mechanistic 6.0 nM (IC50) ChEMBL
CHEMBL2376519 ↓ inhibits Mechanistic 81.82 nM (IC50) ChEMBL
CHEMBL4456791 ↓ inhibits Mechanistic 1900.0 nM (IC50) ChEMBL
CHEMBL4473371 ↓ inhibits Mechanistic 3.07 nM (Ki) ChEMBL
CHEMBL4518438 ↓ inhibits Mechanistic 46.5 nM (IC50) ChEMBL
CHEMBL4516351 ↓ inhibits Mechanistic 57.4 nM (IC50) ChEMBL
CHEMBL4447626 ↓ inhibits Mechanistic 87.7 nM (IC50) ChEMBL
CHEMBL4544630 ↓ inhibits Mechanistic 96.6 nM (IC50) ChEMBL
CHEMBL4514263 ↓ inhibits Mechanistic 62.3 nM (IC50) ChEMBL
CHEMBL4518992 ↓ inhibits Mechanistic 57.7 nM (IC50) ChEMBL
CHEMBL5176418 ↓ inhibits Mechanistic 120.0 nM (Ki) ChEMBL
DONEPEZIL ↓ inhibits Mechanistic 32.36 nM (IC50) ChEMBL

Showing top 40 of 57 agents ranked by evidence tier.

FCGRT

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EFGARTIGIMOD ALFA ↓ inhibits Mechanistic DGIdb
BATOCLIMAB ? unclear Mechanistic DGIdb
DX-2507 ? unclear Mechanistic DGIdb
ZILTIVEKIMAB ? unclear Mechanistic DGIdb
NIPOCALIMAB ↓ inhibits Mechanistic DGIdb
ROZANOLIXIZUMAB ↓ inhibits Mechanistic DGIdb
ORILANOLIMAB ↓ inhibits Mechanistic DGIdb
CHEMBL508564 ↓ inhibits Mechanistic 1400.0 nM (IC50) ChEMBL
CHEMBL508411 ↓ inhibits Mechanistic 36000.0 nM (IC50) ChEMBL
CHEMBL505689 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
CHEMBL524509 ↓ inhibits Mechanistic 31000.0 nM (IC50) ChEMBL
CHEMBL503730 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL526683 ↓ inhibits Mechanistic 67000.0 nM (IC50) ChEMBL
CHEMBL207341 ↓ inhibits Mechanistic 24000.0 nM (IC50) ChEMBL
CHEMBL451944 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL506507 ↓ inhibits Mechanistic 40000.0 nM (IC50) ChEMBL
CHEMBL509774 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
PSYCIEGHIDGIYCFNA ↓ inhibits Mechanistic 49000.0 nM (IC50) ChEMBL
CHEMBL503219 ↓ inhibits Mechanistic 64000.0 nM (IC50) ChEMBL
CHEMBL499790 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
CHEMBL444514 ↓ inhibits Mechanistic 20000.0 nM (Kd) ChEMBL
CHEMBL525216 ↓ inhibits Mechanistic 34000.0 nM (Kd) ChEMBL
CHEMBL499664 ↓ inhibits Mechanistic 69000.0 nM (IC50) ChEMBL
CHEMBL499402 ↓ inhibits Mechanistic 57000.0 nM (IC50) ChEMBL
CHEMBL506644 ↓ inhibits Mechanistic 48000.0 nM (IC50) ChEMBL
CHEMBL525265 ↓ inhibits Mechanistic 420.0 nM (IC50) ChEMBL
CHEMBL503983 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL504423 ↓ inhibits Mechanistic 2100.0 nM (IC50) ChEMBL
CHEMBL525614 ↓ inhibits Mechanistic 1600.0 nM (IC50) ChEMBL
CHEMBL505899 ↓ inhibits Mechanistic 900.0 nM (IC50) ChEMBL
CHEMBL511030 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL446284 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL510716 ↓ inhibits Mechanistic 1700.0 nM (IC50) ChEMBL
CHEMBL504919 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL499766 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL524659 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL526861 ↓ inhibits Mechanistic 18000.0 nM (IC50) ChEMBL
CHEMBL506647 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL504226 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL507004 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL

Showing top 40 of 120 agents ranked by evidence tier.

IL6R

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SATRALIZUMAB ? unclear Mechanistic DGIdb
SIRUKUMAB ? unclear Mechanistic DGIdb
TOCILIZUMAB ↓ inhibits Mechanistic DGIdb
SARILUMAB ? unclear Mechanistic DGIdb
PAREGORIC ? unclear Mechanistic DGIdb
VOBARILIZUMAB ? unclear Mechanistic DGIdb
LEVILIMAB ↓ inhibits Mechanistic DGIdb
BAZEDOXIFENE ? unclear Mechanistic DGIdb
PROTEASOME INHIBITOR ? unclear Mechanistic DGIdb
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic DGIdb
THALIDOMIDE ? unclear Mechanistic DGIdb
IL-6 ↑ activates Mechanistic DGIdb
FLUOROURACIL ? unclear Mechanistic DGIdb

LRP4

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

MS4A1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RITUXIMAB ~ modulates Mechanistic DGIdb
UBLITUXIMAB ~ modulates Mechanistic DGIdb
VELTUZUMAB ? unclear Mechanistic DGIdb
OFATUMUMAB ~ modulates Mechanistic DGIdb
OBINUTUZUMAB ~ modulates Mechanistic DGIdb
OCARATUZUMAB ~ modulates Mechanistic DGIdb
TOSITUMOMAB ~ modulates Mechanistic DGIdb
TISAGENLECLEUCEL ? unclear Mechanistic DGIdb
IODINE-131-TOSITUMOMAB ? unclear Mechanistic DGIdb
EPCORITAMAB ? unclear Mechanistic DGIdb
GLOFITAMAB ~ modulates Mechanistic DGIdb
TRU-015 ~ modulates Mechanistic DGIdb
ODRONEXTAMAB ? unclear Mechanistic DGIdb
OCRELIZUMAB ~ modulates Mechanistic DGIdb
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic DGIdb
DI-LEU16-IL2 ? unclear Mechanistic DGIdb
MONOCLONAL ANTIBODY 1F5 ? unclear Mechanistic DGIdb
MT-3724 ~ modulates Mechanistic DGIdb
MOSUNETUZUMAB ? unclear Mechanistic DGIdb
PLAMOTAMAB ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD20 CAR TRANSDUCED CD4/CD8 ENRICHED T-CELLS MB-CART20.1 ? unclear Mechanistic DGIdb
FBT-A05 ~ modulates Mechanistic DGIdb
YTTRIUM Y 90 IBRITUMOMAB TIUXETAN ~ modulates Mechanistic DGIdb
BSAB (CD89-CD20) ? unclear Mechanistic DGIdb
LFB-R603 ~ modulates Mechanistic DGIdb
DIVOZILIMAB ? unclear Mechanistic DGIdb
IBRITUMOMAB TIUXETAN ? unclear Mechanistic DGIdb
TOSITUMOMAB 131I ~ modulates Mechanistic DGIdb

MUSK

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
HESPERADIN ↓ inhibits Mechanistic DGIdb
COMPOUND 1D [PMID: 21493067] ↓ inhibits Mechanistic DGIdb
NIPOCALIMAB ? unclear Mechanistic DGIdb
ROZANOLIXIZUMAB ? unclear Mechanistic DGIdb
COMPOUND 25 [PMID: 17935989] ↓ inhibits Mechanistic DGIdb
LINIFANIB ↓ inhibits Mechanistic 10.0 nM (Kd) ChEMBL
AST-487 ↓ inhibits Mechanistic 3.1 nM (Kd) ChEMBL
DEFOSBARASERTIB ↓ inhibits Mechanistic 1500.0 nM (Kd) ChEMBL
DORAMAPIMOD ↓ inhibits Mechanistic 1900.0 nM (Kd) ChEMBL
DOVITINIB ↓ inhibits Mechanistic 830.0 nM (Kd) ChEMBL
SORAFENIB ↓ inhibits Mechanistic 130.0 nM (Kd) ChEMBL
STAUROSPORINE ↓ inhibits Mechanistic 86.0 nM (Kd) ChEMBL
SU-014813 ↓ inhibits Mechanistic 100.0 nM (Kd) ChEMBL
SUNITINIB ↓ inhibits Mechanistic 490.0 nM (Kd) ChEMBL
TOZASERTIB ↓ inhibits Mechanistic 300.0 nM (Kd) ChEMBL
QUIZARTINIB ↓ inhibits Mechanistic 1000.0 nM (Kd) ChEMBL
CHEMBL1774056 ↓ inhibits Mechanistic 224.0 nM (IC50) ChEMBL
CRIZOTINIB ↓ inhibits Mechanistic 230.0 nM (Kd) ChEMBL
TAE-684 ↓ inhibits Mechanistic 260.0 nM (Kd) ChEMBL
CHEMBL1908395 ↓ inhibits Mechanistic 5600.0 nM (Kd) ChEMBL
NINTEDANIB ↓ inhibits Mechanistic 590.0 nM (Kd) ChEMBL
FORETINIB ↓ inhibits Mechanistic 13.0 nM (Kd) ChEMBL
KW-2449 ↓ inhibits Mechanistic 200.0 nM (Kd) ChEMBL
FEDRATINIB ↓ inhibits Mechanistic 800.0 nM (Kd) ChEMBL
R-406 ↓ inhibits Mechanistic 100.0 nM (Kd) ChEMBL
LESTAURTINIB ↓ inhibits Mechanistic 4100.0 nM (Kd) ChEMBL
CHEMBL3422092 ↓ inhibits Mechanistic 690.0 nM (IC50) ChEMBL
CHEMBL4553037 ↓ inhibits Mechanistic 530.0 nM (Kd) ChEMBL
PF-06273340 ↓ inhibits Mechanistic 53.0 nM (IC50) ChEMBL
CHEMBL4792981 ↓ inhibits Mechanistic 110.0 nM (Kd) ChEMBL
TP-030-1 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
TP-030-2 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
TP-030n ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
MILREBRUTINIB ↓ inhibits Mechanistic 306.0 nM (IC50) ChEMBL
CHEMBL5423601 ↓ inhibits Mechanistic 12.83 nM (IC50) ChEMBL
CHEMBL5559304 ↓ inhibits Mechanistic 257.0 nM (IC50) ChEMBL
BMS-754807 ↓ inhibits Mechanistic 6000.0 nM (Kd) ChEMBL

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Myasthenia Gravis (MG): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis Recruiting
NCT06359041 Phase: 1/2 Started: 2024-12-17

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis

ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis Recruiting
NCT07673627 Phase: 2 Started: 2026-07-10

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects an...

A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG) Active Not Recruiting
NCT06456580 Phase: 3 Started: 2024-07-17

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod Recruiting
NCT07284420 Phase: 2 Started: 2025-12-19

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects an...

A Study to Evaluate the Efficacy and Safety of Maintenance Ublituximab Following Induction With Efgartigimod Administration in Participants With Myasthenia Gravis (MG) Recruiting
NCT07673744 Phase: 2 Started: 2026-10-01

The primary purpose of this study is to evaluate the efficacy of ublituximab in adult participants with MG responding to treatment with efgartigimod.

Zilucoplan for Severe gMG Exacerbations Recruiting
NCT07215949 Phase: 3 Started: 2026-01-20

This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.

Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG) Active Not Recruiting
NCT04146051 Phase: 2 Started: 2019-12-04

This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis

KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis Recruiting
NCT06193889 Phase: 2/3 Started: 2024-08-28

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis

Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis Active Not Recruiting
NCT06106672 Phase: 1/2 Started: 2024-05-30

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.

An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis Completed
NCT04980495 Phase: 3 Started: 2021-12-16

The purpose of this open-label study is to investigate the efficacy, safety, and tolerability of a continuous regimen of efgartigimod compared with a cyclic regimen in participants with Generalized Myasthenia Gravis (gMG). Participants will receive efgartigimod throughout the study. The participants...

Efficacy and Safety of HN2302 in Refractory Myasthenia Gravis(MG) Recruiting
NCT07413835 Phase: N/A Started: 2026-03-17

This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.

Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis Completed
NCT05451212 Phase: 1 Started: 2022-11-23

Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various...

Subcutaneous Immunoglobulin for Myasthenia Gravis Completed
NCT04728425 Phase: 2 Started: 2020-08-28

This is a prospective open-label, randomized, parallel arm clinical trial. The primary objective of the study is to evaluate the safety and efficacy of Cuvitru 20% subcutaneous immunoglobulin in patients with myasthenia gravis (MG). The secondary objective is to evaluate patient preferences and effe...

A Study Evaluating the Safety and Efficacy of Rituximab in Patients With Myasthenia Gravis Completed
NCT02950155 Phase: 3 Started: 2016-10-16

A randomized, double-blind, placebo-controlled multicenter study evaluating the safety and efficacy of Rituximab (Mabthera®) in patients with new onset generalized myasthenia gravis (MG).

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG Completed
NCT03304054 Phase: 3 Started: 2018-04-18

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.

Agents Found By Disease-Level Search

Searched directly from “Myasthenia Gravis (MG)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

48 agents shown (approved or with trial evidence) — 43 not found by the gene-target search above.

efgartigimod IVNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT04735432 NCT04833894 NCT07294170

EfgartigimodNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT07217587 NCT06299748 NCT06298565

efgartigimod PH20 SCNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04735432 NCT06392386

Rozanolixizumab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT04124965 NCT07246564

TelitaciceptNOT in gene-target list

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06456580 NCT05737160

CABA-201NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06359041 NCT06359041

Nipocalimab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT04951622 NCT07217587

CD19-BCMA Targeted CAR-T Dose 2NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06371040 NCT06371040

Amifampridine PhosphateNOT in gene-target list

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01377922 NCT03304054

Empasiprubart IVNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07673627 NCT07294170

Efgartigimod PH20 SC PFSNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07673627 NCT07294170

Ravulizumab

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT05644561 NCT06967480

Eculizumab

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03759366

IM-101 Part ANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07250750

IM-101 Part BNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07250750

Nipocalimab SC-LIVNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04951622

CD19-BCMA Targeted CAR-T Dose 1NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06371040

MedlinkNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06256172

MuSK-CAARTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05451212

PovetaciceptNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07501702

ClaseprubartNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06282159

subcutaneous immunoglobulin (SCIG)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04728425

intravenous immunoglobulin + subcutaneous immunoglobulin (SCIG)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04728425

efgartigimod administrationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06765161

CD19/BCMA-targeted CAR-T cellsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07337785

Lymphodepleting ConditioningNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07337785

Control GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06624345

zolacabtagene autoleucel (CC-97540)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06220201

FludarabineNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06220201

CyclophosphamideNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06220201

Tacrolimus capsuleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01325571

BelimumabNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01480596

IGIV-CNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02413580

Mycophenolate MofetilNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03490539

AzathioprineNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03490539

gefurulimab (ALXN1720)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05556096

rozanolixizumab (UCB7665)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT03052751

Serum Bactericidal AssayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06906692

Heart Rate Variability,HRV - ANSWatchNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06754020

Tocilizumab Injectable ProductNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05067348

BAFF-R CARTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07022197

Transcervical ThymectomyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03825185

Transsternal ThymectomyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03825185

Rituximab

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02950155

Sodium Chloride solutionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02950155

Social media recruitmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06866483

Follow-upNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06866483

HBM9161 Injection (680mg and 340 mg)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04346888

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.