Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

Address: Clinic for Special Children, Strasburg, PA, USA. [email protected].; Penn Medicine-Lancaster General Hospital, Lancaster, PA, USA. [email protected].; Departments of Pediatrics and Molecular, Cell & Cancer Biology, University of Massachusetts School of Medicine, Worcester, MA, USA. [email protected].; Department of Neurology, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, New South Wales, Australia.; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Department of Neurology, The Ohio State University, Columbus, OH, USA.; Department of Paediatrics, MDUK Oxford Neuromuscular Centre, Oxford, UK.; Neuromuscular Reference Center, Department of Pediatrics, CHU & University of Liège, Liège, Belgium.; Departments of Pediatrics, Neurology & Neurosurgery, Montreal Children's Hospital, McGill University Health Centre, Montreal, Quebec, Canada.; Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA.; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.; Washington University School of Medicine, St. Louis, MO, USA.; Division of Pediatric Neurology, Columbia University Medical Center, New York, NY, USA.; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.; Department of Neurology, Helen DeVos Children's Hospital, Grand Rapids, MI, USA.; Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA.; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; Translational Medicine, Novartis Institutes for BioMedical Research, Cambridge, MA, USA.