Congenital myasthenic syndrome Advocacy Hub | UniteRare

At a Glance

Live from database

🔬

4Recruiting trials

🩺

7Specialists

Regulatory Watchboard

10 events

Oct 2026hiPSC-CMs-loaded Chitosan Patch to Treat Severe Chronic Ischemic Heart Disease

HELP Therapeutics Co., Ltd. — PHASE1, PHASE2

TrialNOT YET RECRUITING

Mar 2026A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase Ⅱ Clinical Study to Evaluate the Efficacy and Safety of CMS-D001 in Adult Patients With Moderate to Severe Atopic Dermatitis

Dermavon Holdings Limited — PHASE2

TrialNOT YET RECRUITING

Mar 2026A Clinical Study Evaluating the Efficacy and Safety of CMS-D001 Tablets in the Treatment of Adult Patients With Moderate to Severe Plaque-type Psoriasis

Dermavon Holdings Limited — PHASE2, PHASE3

TrialNOT YET RECRUITING

Feb 2026Phase II Study of CMS-D002 Capsule for Uterine Fibroids With Menorrhagia

Shenzhen Kangzhe Biotechnology Co., Ltd. — PHASE2

TrialRECRUITING

Oct 2025Healthcare Integrity & Anti-Fraud Enforcement

Truway Health, Inc.

TrialENROLLING BY INVITATION

Jul 2025Erythrocytapheresis for Chronic Mountain Sickness

Third Military Medical University — NA

TrialRECRUITING

May 2025A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes

National Institute of Neurological Disorders and Stroke (NINDS)

TrialRECRUITING

Mar 2025IPSC-CMs Combined with LVAD or CABG for the Treatment of Heart Failure

HELP Therapeutics Co., Ltd. — PHASE1, PHASE2

TrialNOT YET RECRUITING

Jan 2025Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution

The Foundation for Orthopaedics and Regenerative Medicine — PHASE1

TrialRECRUITING

Sep 2024A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

argenx — PHASE1

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Congenital myasthenic syndrome.
Check the disease page for updates →

Clinical Trial Landscape

4 active trials

2Phase 1

2Unknown

4Total recruiting

Search clinical trials for Congenital myasthenic syndrome →

Recent News & Research

No recent news articles indexed yet for Congenital myasthenic syndrome.
Search PubMed for Congenital myasthenic syndrome →

Browse all Congenital myasthenic syndrome news →

Specialist Network

Top 6 by expertise

Andrew G Engel, MD

Mayo Clinic

Score 261 trial1 pub

Laurent Servais, MD

Centre Hospitalier Universitaire de Liege

Score 226 trials1 pub

Christopher Grunseich, M.D.

National Institute of Neurological Disorders and Stroke (NINDS)

📍 Bethesda, Maryland

Score 85 trials

Thomas Crawford, M.D.

Johns Hopkins Pediatric Neurology

Score 61 trial

Laurent SERVAIS, MD, PhD

institut myologie

Score 21 trial

Simon Edvardson

Hadassah Medical Organization

Score 01 trial

View all Congenital myasthenic syndrome specialists →

Quick Actions

📄

View Disease Page

Full profile, treatments, community

🔬

Search Clinical Trials

Find recruiting studies now

🩺

Find Specialists

Ranked by expertise & trials

💰

Financial Assistance

Copay cards, PAPs, grants

📰