X-linked myotubular myopathy

Clinical trials for X-linked myotubular myopathy explained in plain language.

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Gene therapy trial for rare muscle disease halted after severe side effects

Disease control Ongoing

This trial tested a gene therapy called AT132 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that causes severe breathing problems. The therapy aimed to deliver a healthy copy of the MTM1 gene to improve muscle function. However, the study was st…

Matched conditions: X-LINKED MYOTUBULAR MYOPATHY

Phase 2/3 • Sponsor: Astellas Gene Therapies • Aim: Disease control

Last updated Sep 05, 2026 00:00 UTC

X-linked myotubular myopathy

Get alerted when new X-linked myotubular myopathy trials appear

Gene therapy trial for rare muscle disease halted after severe side effects