One shot of gene therapy aims to fix a rare Muscle-Weakening disease
Cure
Not yet recruiting
Researchers are testing AMP-101, an experimental gene therapy, in people with DOK7 congenital myasthenic syndrome, a rare inherited disorder that causes muscle weakness and exercise intolerance. About 4 participants with moderate to severe DOK7 CMS will receive a single intraveno…
Phase 1 • Sponsor: Amplo Biotechnology • Aim: Cure
Last updated Sep 21, 2026 23:00 UTC