One-Time gene therapy aims to halt rare muscle disease
NCT ID NCT06246513
First seen Mar 27, 2026 · Last updated Apr 24, 2026 · Updated 6 times
Summary
This study tests a single dose of gene therapy (SRP-9003) 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. Both walkers and non-walkers can join. The treatment is not expected to be a cure, but may slow disease progression.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital of The King's Daughter
Norfolk, Virginia, 23507, United States
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Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Hospital Sant Joan de Deu
Barcelona, 8950, Spain
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NMRC Gent (UZ Gent)
Ghent, 9000, Belgium
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Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
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Newcastle University
Newcastle upon Tyne, NE1 3BZ, United Kingdom
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen, North Rhine-Westphalia, 45147, Germany
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University Hospital Leuven (UZ Leuven)
Leuven, Vlaams Brabant, 3000, Belgium
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University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
Conditions
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