One-Time gene shot aims to halt rare muscle disease
NCT ID NCT06246513
First seen Mar 27, 2026 · Last updated May 24, 2026 · Updated 13 times
Summary
This study tests a single dose of SRP-9003 gene therapy in 17 people with limb-girdle muscular dystrophy 2E/R4, a rare genetic muscle-weakening disease. The goal is to see if the therapy can restore a missing protein in muscle cells and improve function. Both walkers and non-walkers are included, and the study is currently active but not recruiting.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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