Intrathecal onasemnogene abeparvovec (OAV101 IT) for patients with spinal muscular atrophy (SMA): Phase 3, randomized, sham-controlled, double-blind steer study
Year: 2025
Type: article
Language: en
Topic: Neurogenetic and Muscular Disorders Research
Authors and affiliations:
raw_author_name: Crystal Proud
raw_affiliation_strings: Children's Hospital of the King's Daughters, N/a, Norfolk, United States of America
raw_author_name: Dũng Chí Vũ
raw_affiliation_strings: Hanoi University of Public Health, Pediatric Division, Department of Clinical Medicine, Hanoi, Viet Nam
Vietnam National Children's Hospital, Center for Genetics, Molecular Therapy, Metabolism and Endocrinology, Department of Clinical Genetics/genomics and Molecular Therapy, Hanoi, Viet Nam
raw_author_name: Jo Wilmshurst
raw_affiliation_strings: Red Cross War Memorial Children's Hospital, University of Cape Town, Pediatric Neurology, Department of Pediatrics and Child Health, Cape Town, South Africa
raw_author_name: Oranee Sanmaneechai
raw_affiliation_strings: Siriraj Hospital, Mahidol University, Center of Research Excellent In Neuromuscular Disease, Faculty of Medicine, Bangkok, Thailand
Siriraj Hospital, Mahidol University, Division of Neurology, Department of Pediatrics, Faculty of Medicine, Bangkok, Thailand
raw_author_name: Sheffali Gulati
raw_affiliation_strings: AIIMS, Centre of Excellence & Advanced Research for Childhood Neuro-developmental Disorders & Child Neurology Division, Department of Pediatrics, New Delhi, India
raw_author_name: Hui Xiong
raw_affiliation_strings: Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Department of Neurology, Beijing, China
Peking University First Hospital, Children's Medical Center, Beijing, China
raw_author_name: Hugo Ceja Moreno
raw_affiliation_strings: Hospital Civil de Guadalajara Fray Antonio Alcalde, N/a, Guadalajara, Mexico
raw_author_name: Stacey Kiat Hong Tay
raw_affiliation_strings: Yong Loo Lin School of Medicine, National University of Singapore, Division of Pediatric Neurology, Department of Pediatrics, Singapore, Singapore
raw_author_name: Meow-Keong Thong
raw_affiliation_strings: Universiti Tunku Abdul Rahman, M. Kandiah Faculty of Medicine and Health Sciences, Kajang, Selangor, Malaysia
University of Malaya, Genetic Medicine Unit, Department of Pediatrics, Faculty of Medicine, Kuala Lumpur, Malaysia
raw_author_name: Alfred Peter Born
raw_affiliation_strings: Rigshospitalet, Copenhagen University Hospital, N/a, Copenhagen, Denmark
raw_author_name: Adriana Banzzatto Ortega
raw_affiliation_strings: Hospital Pequeno Príncipe, N/a, Curitiba, Brazil
raw_author_name: Yuh-Jyh Jong
raw_affiliation_strings: Kaohsiung Medical University Hospital, Kaohsiung Medical University, Departments of Pediatrics and Laboratory Medicine, and Translational Research Center of Neuromuscular Diseases, Kaohsiung, Taiwan
Kaohsiung Medical University, Graduate Institute of Clinical Medicine, Kaohsiung, Taiwan
National Yang Ming Chiao Tung University, Department of Biological Science and Technology, Hsinchu, Taiwan
raw_author_name: Mohammad Al-Muhaizea
raw_affiliation_strings: King Faisal Specialist Hospital & Research Centre, Neuromuscular Integrated Practice Unit, Neuroscience Centre, Riyadh, Saudi Arabia
raw_author_name: Anna Lee
raw_affiliation_strings: Novartis Pharmaceuticals, Clinical Development / Neuroscience & Gene Therapy, East Hanover, United States of America
raw_author_name: Jeannie Visootsak
raw_affiliation_strings: Novartis Pharmaceuticals, Neuroscience and Gene Therapies, East Hanover, United States of America
raw_author_name: Sitra Tauscher-Wisniewski
raw_affiliation_strings: Novartis Gene Therapies, Inc., N/a, Bannockburn, United States of America
raw_author_name: Iulian Alecu
raw_affiliation_strings: Novartis Pharmaceuticals, Global Safety Program, Basel, Switzerland
raw_author_name: Rutvick Parlikar
raw_affiliation_strings: Novartis, Biostatistics, Hyderabad, India
raw_author_name: Richard Finkel
raw_affiliation_strings: St. Jude Children's Research Hospital, Center for Experimental Neurotherapeutics, Memphis, United States of America