LIMB-GIRDLE MUSCULAR DYSTROPHY, TYPE 2E

Clinical trials for LIMB-GIRDLE MUSCULAR DYSTROPHY, TYPE 2E explained in plain language.

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Terminated 1

First human test of gene therapy for rare muscle disorder

Disease control Terminated

This was an early-stage study to test the safety and initial effects of an experimental gene therapy called SRP-9003 for people with a rare, inherited muscle-wasting disease called LGMD2E. The therapy aimed to deliver a working copy of a faulty gene directly into the body via an …

Matched conditions: LIMB-GIRDLE MUSCULAR DYSTROPHY, TYPE 2E

Phase: PHASE1, PHASE2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control

Last updated Mar 09, 2026 14:24 UTC

LIMB-GIRDLE MUSCULAR DYSTROPHY, TYPE 2E

Get alerted when new LIMB-GIRDLE MUSCULAR DYSTROPHY, TYPE 2E trials appear

First human test of gene therapy for rare muscle disorder