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 a severe, life-threatening form of spinal muscular atrophy (SMA Type 1). The goal was to see if a one-time intravenous infusion could help babies achieve major milestones like sitting independently and surviving without permanent breathing support. The trial followed 33 babies to measure their development and safety over 18 months.
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