Gene therapy hope for toddlers with duchenne muscular dystrophy
NCT ID NCT06128564
First seen Nov 01, 2025 · Last updated Apr 25, 2026 · Updated 28 times
Summary
This study tests a gene therapy called delandistrogene moxeparvovec in children under 4 with Duchenne muscular dystrophy, a severe muscle-weakening disease. The goal is to see if the treatment is safe and helps produce a key muscle protein. Thirteen children will be followed for about 5 years to monitor side effects and protein levels.
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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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Chr de La Citadelle
Liège, 3500, Belgium
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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PU A. Gemelli, Università Cattolica del Sacro Cuore
Rome, Lazio, 00168, Italy
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Universitätsklinikum Essen
Essen, 45147, Germany
Conditions
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