LIMB-GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB-GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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Gene therapy trial for rare muscle disease halted after early phase
Disease control TerminatedThis early-stage study aimed to test the safety of a single-dose gene therapy called SRP-9004 for a rare, inherited muscle-wasting disorder. It involved a small group of participants, some who could walk and some who could not, to see if the treatment was safe and if it could inc…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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Early gene therapy trial for rare muscle disease halted
Disease control TerminatedThis early-stage study tested whether a gene therapy called SRP-6004 could safely deliver a working gene to people with limb girdle muscular dystrophy type 2B, a rare genetic muscle disorder. The trial involved just 2 participants who could still walk and had specific genetic mut…
Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC