LIMB-GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB-GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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First human test of gene therapy for rare muscle disease
Disease control OngoingThis early-stage study is testing a single dose of an experimental gene therapy called SRP-9003 in six people with a rare genetic muscle disease called limb girdle muscular dystrophy type 2E/R4. The main goals are to check if the treatment is safe and to see if it increases a nee…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Apr 02, 2026 07:26 UTC
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Gene therapy trial aims to restore muscle protein in rare muscular dystrophy
Disease control OngoingThis Phase 3 study is testing a single-dose gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E/R4. The therapy aims to deliver a working copy of the beta-sarcoglycan gene to help muscles produce a crucial protein they lack. The study will measure …
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Scientists launch global effort to map the journey of a rare muscle disease
Knowledge-focused OngoingThis study aims to understand how four specific types of limb-girdle muscular dystrophy (LGMD) change over time. It will follow 205 participants, aged 4 and older, for up to 5 years to track their mobility, lung function, and other symptoms. The goal is to gather detailed informa…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Apr 02, 2026 07:26 UTC
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Scientists map muscle decline in rare disease to pave way for future treatments
Knowledge-focused OngoingThis study aims to understand how limb-girdle muscular dystrophy type 2A (LGMD2A) progresses over two years. Researchers will closely monitor 25 patients to measure changes in their muscle strength, movement abilities, and quality of life. The goal is to gather detailed informati…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:37 UTC