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Not yet finished but already full! 1 Completed 1

Gene therapy trial offers hope for rare muscle disease

Disease control Ongoing

This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.

Matched conditions: LGMD

Phase 1/2 • Sponsor: AskBio Inc • Aim: Disease control

Last updated Aug 28, 2026 00:00 UTC

LGMD

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Gene therapy trial offers hope for rare muscle disease