LGMD2I
Clinical trials for LGMD2I explained in plain language.
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Gene therapy trial hopes to slow rare muscle disease
Disease control Recruiting nowThis study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9) that causes progressive weakness. The main goal is to check safety, not yet to prove it works. Participants must be able to walk 10 meters in under 30 secon…
Matched conditions: LGMD2I
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated May 13, 2026 16:03 UTC
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Major study aims to better measure muscle decline in rare diseases
Knowledge-focused Recruiting nowThis 24-month observational study will follow up to 1000 people with certain types of muscular dystrophy (LGMD, DM2, or late-onset Pompe disease) to see how well specific muscle function tests work over time. Participants will be between 6 and 50 years old and must have a genetic…
Matched conditions: LGMD2I
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated May 15, 2026 11:55 UTC