LGMD2I
Clinical trials for LGMD2I explained in plain language.
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First human test of gene therapy for rare muscle disease begins
Disease control Recruiting nowThis study is testing the safety of a single-dose gene therapy called AB-1003 in adults with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle disorder. Researchers will give increasing doses to small groups of participants to check for side effects. The goal is to…
Matched conditions: LGMD2I
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Massive study launches to map the course of rare muscle diseases
Knowledge-focused Recruiting nowThis study aims to better understand how several rare types of muscular dystrophy progress over time. Researchers will observe up to 1,000 participants for two years, tracking their strength, movement, and quality of life using standard tests and questionnaires. The goal is to ga…
Matched conditions: LGMD2I
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:31 UTC