33
Participants
Start Date
August 16, 2018
Primary Completion Date
September 11, 2020
Study Completion Date
September 11, 2020
Onasemnogene Abeparvovec-xioi
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken β-actin-hybrid promoter (CB).
University Hospital Ghent Neuromuscular reference center, Ghent
Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires, Liège
Hôpital Armand Trousseau, Paris
Istituto Gianninia Gaslini, Genova
"Policlinico G. Martino", Messina
Carlo Besta Neurological Research Institute, Milan
University of Milan, Milan
Policlinico Gemelli, Rome
Great Ormond Street Hospital for Children, London
The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle, Newcastle upon Tyne
Lead Sponsor
Novartis Gene Therapies
INDUSTRY