Single infusion offers hope for babies with devastating muscle disease
NCT ID NCT03461289
Summary
This study tested a one-time gene therapy for babies under 6 months old with spinal muscular atrophy type 1, a severe genetic disease that weakens muscles. The therapy aimed to deliver a working copy of a missing gene through a single intravenous infusion. Researchers measured whether treated babies could sit independently and survive without permanent breathing support.
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Contacts and locations
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Locations
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Carlo Besta Neurological Research Institute
Milan, Italy
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Great Ormond Street Hospital for Children
London, United Kingdom
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Hôpital Armand Trousseau
Paris, France
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Istituto Gianninia Gaslini
Genova, Italy
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Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires
Liège, Belgium
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Policlinico "G. Martino"
Messina, Italy
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Policlinico Gemelli
Rome, Italy
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The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle
Newcastle upon Tyne, United Kingdom
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University Hospital Ghent Neuromuscular reference center
Ghent, Belgium
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University of Milan
Milan, Italy
Conditions
Explore the condition pages connected to this study.