Research.Patient.info
Congenital Myasthenic Syndrome research hub
2 indexed studies · 2 currently recruiting
- recruitingPHASE1
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
This study is testing a new medicine, ARGX-119, for adults with a specific type of muscle weakness called DOK7-Congenital Myasthenic Syndrome (CMS). It's looking at how safe the medicine is, if it causes side effects, and if it helps people feel better.
United States · Canada · France - recruiting
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
This study looks at people with a rare muscle condition called Congenital Myasthenic Syndrome (CMS) caused by specific gene problems. It aims to understand how the condition naturally affects patients' daily lives by checking their symptoms and quality of life over several visits.
United States · Austria · Belgium