One-Time gene therapy aims to halt rare muscle disease

NCT ID NCT06246513

Ongoing Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the study is now active but not recruiting.

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2E limb-girdle muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

LIMB GIRDLE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Children's Hospital of The King's Daughter

Norfolk, Virginia, 23507, United States
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Hospital Sant Joan de Deu

Barcelona, 8950, Spain
NMRC Gent (UZ Gent)

Ghent, 9000, Belgium
Nationwide Childrens Hospital

Columbus, Ohio, 43205, United States
Newcastle University

Newcastle upon Tyne, NE1 3BZ, United Kingdom
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum

Essen, North Rhine-Westphalia, 45147, Germany
University Hospital Leuven (UZ Leuven)

Leuven, Vlaams Brabant, 3000, Belgium
University of California, San Diego-Altman Clinical and Translational Research Institute

La Jolla, California, 92037, United States

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