Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

计划入组 21 人 · 首次发布于 2010 年 9 月 16 日

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

计划入组 20 人 · 首次发布于 2015 年 9 月 29 日

3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)

首次发布于 2009 年 4 月 1 日

Pyridostigmine(Mestinon, Regonol)

C₉H₁₃N₂O₂ · Rx

AAV9 based ChAT gene therapy (congenital myasthenic syndrome)

congenital hyperinsulinism therapeutic

InspireMD

VentriPoint Diagnostics Ltd

ALX Oncology

United States

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

NIH Funding Cuts Under Trump Administration Threaten Early-Stage Biopharma Innovation