Treatment

ASP2957 clinical trials

1 indexed studies · 0 currently recruiting

AuthorisedPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants with Invasive Ventilator-dependent X-linked Myotubular Myopathy
This study is testing a new medicine, ASP2957, for boys and men with X-linked myotubular myopathy. It aims to see if the medicine is safe, what side effects it might cause, and if it could help improve their condition, especially with breathing support.
For: X-linked myotubular myopathy
France