Astellas Gene Therapies
Clinical trials sponsored by Astellas Gene Therapies, explained in plain language.
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Experimental gene therapy aims to help boys with rare muscle disease breathe easier
Disease control Recruiting nowThis early-phase trial tests a new gene therapy called ASP2957 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a modified virus. Nine boys up t…
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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New study aims to uncover hidden liver risks in rare muscle disease
Knowledge-focused Recruiting nowThis study follows about 50 boys under 18 with X-linked myotubular myopathy (XLMTM), a rare genetic muscle condition, to see how often they develop liver problems like cholestasis. No drugs are given—researchers simply collect health data, including liver scans, over one year. Th…
Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Jul 01, 2026 00:00 UTC