X-LINKED MYOTUBULAR MYOPATHY
Clinical trials for X-LINKED MYOTUBULAR MYOPATHY explained in plain language.
Never miss a new study
Get alerted when new X-LINKED MYOTUBULAR MYOPATHY trials appear
Sign up with your email to follow new studies for X-LINKED MYOTUBULAR MYOPATHY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
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…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
-
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…
Matched conditions: X-LINKED MYOTUBULAR MYOPATHY
Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Jul 01, 2026 00:00 UTC