One-Shot gene therapy offers hope for babies with devastating muscle disease
NCT ID NCT03461289
Summary
This study tested a single-dose gene replacement therapy for infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe genetic disease that weakens muscles. The goal was to see if the one-time intravenous treatment could help babies achieve major milestones like sitting independently and improve survival without permanent breathing support. The trial followed 33 infants for 18 months to measure the therapy's effectiveness and safety.
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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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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
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