LIMB GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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Experimental gene therapy tested for rare muscle disease
Disease control TerminatedThis early-stage study tested a single dose of SRP-9004 gene therapy in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, but researchers also looked at whether the therapy could increase a missing muscle …
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 13, 2026 16:03 UTC
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Experimental gene therapy for rare muscle disease halted early
Disease control TerminatedThis study tested 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 check if the treatment was safe and could help muscles work better. Only 2 people took part before the study was stopped ea…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Apr 26, 2026 20:01 UTC