X-LINKED MYOTUBULAR MYOPATHY
Clinical trials for X-LINKED MYOTUBULAR MYOPATHY explained in plain language.
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First-in-Human gene therapy offers hope for babies on ventilators
Disease control Recruiting nowThis is the first time researchers are testing a new gene therapy called ASP2957 in people. It aims to treat a rare, severe muscle disease called X-linked myotubular myopathy (XLMTM) in very young boys who depend on a ventilator to breathe. The study will first find a safe dose a…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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Researchers track hidden liver risks in rare muscle disease
Knowledge-focused Recruiting nowThis study aims to understand how often liver problems occur in boys with X-linked myotubular myopathy (XLMTM), a serious genetic muscle condition. It will follow about 50 boys under 18 for one year, checking their liver health with scans every 6 weeks. The study only collects he…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:43 UTC