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 small study
Disease control TerminatedThis early-phase study tested a single dose of a gene therapy called SRP-9004 in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, and researchers also looked at changes in muscle protein levels and functi…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 26, 2026 05:22 UTC
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Muscular dystrophy gene therapy trial halted after just 2 patients
Disease control TerminatedThis study tested a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a disease that weakens muscles over time. The goal was to see if the treatment was safe when given through a vein. Only 2 people took part before the study was stopped earl…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC