X-Linked Myotubular Myopathy research hub
3 indexed studies · 2 currently recruiting
- recruiting
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
This study looks at liver health in boys under 18 with X-linked myotubular myopathy (XLMTM). It's an observational study, meaning researchers will collect information over about a year to understand liver, gallbladder, and bile duct issues in XLMTM.
United States · Canada · United Kingdom - recruiting
Myotubular and Centronuclear Myopathy Patient Registry
This registry is for people in the UK with myotubular or centronuclear myopathy. It aims to gather important health information to help researchers learn more, identify people for studies, and improve care. You can register online.
United Kingdom - 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.
France