LIMB GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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Gene therapy hope for rare muscular dystrophy
Disease control OngoingThis early-stage study tests a single-dose 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 restore a missing muscle protein. Both walker…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 17, 2026 15:12 UTC
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One-Time gene shot aims to halt rare muscle disease
Disease control OngoingThis study tests a single dose of a gene therapy called SRP-9003 in 17 people with limb-girdle muscular dystrophy 2E/R4, a rare muscle-weakening disease. The treatment delivers a working copy of the faulty gene to muscle cells. Researchers will measure if the therapy increases th…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 12, 2026 13:41 UTC
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New study monitors 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 undergo manual muscle tests and motor assessments. The goal is to better understand the natural course of the disea…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 17, 2026 15:11 UTC
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Scientists track rare muscle disease to unlock future treatments
Knowledge-focused OngoingThis study follows 205 people with certain types of limb-girdle muscular dystrophy (LGMD) to learn how the disease changes over time. Researchers measure muscle strength, walking ability, and lung function for up to 5 years. The goal is to better understand the disease, which may…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated May 17, 2026 15:10 UTC