Gene therapy tested in toddlers with devastating muscle disease
NCT ID NCT06128564
Summary
This study is testing the safety and biological activity of an experimental gene therapy in very young children with Duchenne muscular dystrophy (DMD). It involves 13 children under 4 years old who will receive a single dose of the therapy and be followed for about 5 years. The main goal is to see if the treatment is safe and if it leads to the production of a needed muscle protein.
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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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