LIMB-GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB-GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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One-Time gene shot aims to halt rare muscle disease
Disease control OngoingThis study tests a single dose of SRP-9003 gene therapy in 17 people with limb-girdle muscular dystrophy 2E/R4, a rare genetic muscle-weakening disease. The goal is to see if the therapy can restore a missing protein in muscle cells and improve function. Both walkers and non-walk…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 26, 2026 04:34 UTC
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Gene therapy trial hopes to restore missing muscle protein
Disease control OngoingThis study tests a one-time gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can help muscles produce a missing protein (beta-sarcoglycan). B…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 13:51 UTC
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New study tracks muscle decline in rare muscular dystrophy
Knowledge-focused OngoingThis study follows 25 adults with limb-girdle muscular dystrophy 2A (LGMD2A) to measure how their muscle strength and quality of life change over time. Participants will undergo muscle tests and motor assessments. The goal is to better understand the natural course of the disease…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 26, 2026 05:20 UTC
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Scientists watch rare muscle disease progress in 205 patients
Knowledge-focused OngoingThis study follows 205 people with specific types of limb-girdle muscular dystrophy (LGMD) to learn how the disease affects movement and breathing over several years. Participants are observed and tested regularly, but no new treatment is given. The goal is to better understand t…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated May 26, 2026 04:36 UTC