Gene therapy trial hopes to restore missing muscle protein
NCT ID NCT05876780
First seen Mar 06, 2026 · Last updated May 21, 2026 · Updated 14 times
Summary
This study tests a one-time gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can help muscles produce a missing protein (beta-sarcoglycan). Both walkers and non-walkers can join, but this is an early (Phase 1) trial, so it mainly looks at safety and protein levels, not yet at curing the disease.
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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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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Conditions
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