LIMB GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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Experimental gene therapy for rare muscle disease tested in just 2 people
Disease control TerminatedThis was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing prot…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 14:20 UTC
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Experimental gene therapy tested for rare muscular dystrophy
Disease control TerminatedThis early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:47 UTC