Gene therapy hope for kids with rare Muscle-Wasting disease
NCT ID NCT05973630
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 29 times
Summary
This early-stage study tests a single intravenous gene therapy (ATA-200) in 4 children aged 6-12 with limb girdle muscular dystrophy type 2c (LGMD2C), a rare genetic muscle disease. The main goal is to check safety and side effects, not to cure the disease. Participants must be able to walk and have no antibodies against the virus used to deliver the gene.
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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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Child Health Research Institute
Gainsville, Florida, 32610, United States
Conditions
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