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

Affiliations

• ¹ Clinic for Special Children, Strasburg, PA, USA. [email protected].
• ² Penn Medicine-Lancaster General Hospital, Lancaster, PA, USA. [email protected].
• ³ Departments of Pediatrics and Molecular, Cell & Cancer Biology, University of Massachusetts School of Medicine, Worcester, MA, USA. [email protected].
• ⁴ Department of Neurology, Sydney Children's Hospital Network, Sydney, NSW, Australia.
• ⁵ School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
• ⁶ The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.
• ⁷ National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK.
• ⁸ Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
• ⁹ Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
• ¹⁰ Department of Pediatrics and Department of Neurology, The Ohio State University, Columbus, OH, USA.
• ¹¹ Department of Paediatrics, MDUK Oxford Neuromuscular Centre, Oxford, UK.
• ¹² Neuromuscular Reference Center, Department of Pediatrics, CHU & University of Liège, Liège, Belgium.
• ¹³ Department of Pediatrics, Neurology & Neurosurgery, Montreal Children's Hospital, McGill University, Montreal, QC, Canada.
• ¹⁴ Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA.
• ¹⁵ Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.
• ¹⁶ Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
• ¹⁷ Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.
• ¹⁸ Washington University School of Medicine, St. Louis, MO, USA.
• ¹⁹ Division of Pediatric Neurology, Columbia University Medical Center, New York, NY, USA.
• ²⁰ Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
• ²¹ Department of Neurology, Helen DeVos Children's Hospital, Grand Rapids, MI, USA.
• ²² Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA.
• ²³ Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
• ²⁴ Novartis Gene Therapies, Inc., Bannockburn, IL, USA.
• ²⁵ Translational Medicine, Novartis Institutes for BioMedical Research, Cambridge, MA, USA.