Congenital Myasthenic Syndrome

Trials

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

20 participants · First posted Nov 10, 2025

Completed

NCT01203592

Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

21 participants · First posted Sep 16, 2010

Approved For Marketing

NCT02189720

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

First posted Jul 15, 2014

Drugs

View all 274 →

AAV9 based ChAT gene therapy (congenital myasthenic syndrome)

Small Molecule

Approved

Pyridostigmine(Mestinon, Regonol)

Manufacturer: Valeant pharmaceuticals international, Sandoz canada inc +5 more

Conditions: Congenital Myasthenia (CM), Constipation +5 more

C₉H₁₃N₂O₂ · Rx

congenital hyperinsulinism therapeutic

Organizations

View all 18 →

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

News

View all 3181 →

last year

argenx Advances ARGX-119 to Registrational Study for Ultra-Rare Congenital Myasthenic Syndromes

2 months ago

UC San Diego Researchers Uncover Structural Basis of Congenital Myasthenic Syndromes, Paving Way for Precision Therapies

2 years ago

Trials

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

Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

Drugs

AAV9 based ChAT gene therapy (congenital myasthenic syndrome)

Pyridostigmine(Mestinon, Regonol)

congenital hyperinsulinism therapeutic

Organizations

ALPORT SYNDROME FOUNDATION

International Rett Syndrome Foundation

RETT SYNDROME RESEARCH TRUST

News

argenx Advances ARGX-119 to Registrational Study for Ultra-Rare Congenital Myasthenic Syndromes

UC San Diego Researchers Uncover Structural Basis of Congenital Myasthenic Syndromes, Paving Way for Precision Therapies

MDA Launches Kickstart Program for Ultra-Rare Neuromuscular Disease Gene Therapy Development