X-LINKED MYOTUBULAR MYOPATHY
Clinical trials for X-LINKED MYOTUBULAR MYOPATHY explained in plain language.
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First-in-Human gene therapy trial launches for rare muscle disease in infants
Disease control Recruiting nowThis study tests a new gene therapy called ASP2957 for boys with X-linked myotubular myopathy (XLMTM), a rare and serious muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a harmless virus to he…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated May 22, 2026 14:05 UTC
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New study tracks liver risks in rare muscle disease
Knowledge-focused Recruiting nowThis study follows about 50 boys under 18 with XLMTM, a rare genetic muscle condition, for one year to see how often liver and gallbladder problems occur. Researchers will collect health information and use a special liver scan (Fibroscan) at the start and end of the study. The g…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated May 26, 2026 12:03 UTC