Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial
Year: 2025
Type: article
Language: en
Topic: Neurogenetic and Muscular Disorders Research
Authors and affiliations:
raw_author_name: Crystal M. Proud
raw_affiliation_strings: Children's Hospital of The King's Daughters, Norfolk, VA, USA
raw_author_name: Dũng Chí Vũ
raw_affiliation_strings: Center for Genetics, Molecular Therapy, Metabolism and Endocrinology, Department of Clinical Genetics/Genomics and Molecular Therapy, Vietnam National Children's Hospital, Hanoi, Vietnam
Pediatric Division, Department of Clinical Medicine, Hanoi University of Public Health, Hanoi, Vietnam
raw_author_name: Jo M. Wilmshurst
raw_affiliation_strings: Red Cross War Memorial Children's Hospital, Neuroscience Institute, University of Cape Town, Cape Town, South Africa
raw_author_name: Oranee Sanmaneechai
raw_affiliation_strings: Center of Research Excellent in Neuromuscular Disease, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand
Division of Neurology, Department of Pediatrics, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand
raw_author_name: Sheffali Gulati
raw_affiliation_strings: Centre of Excellence & Advanced Research for Childhood Neuro-developmental Disorders & Child Neurology Division, Department of Pediatrics, AIIMS, New Delhi, India
raw_author_name: Hui Xiong
raw_affiliation_strings: Children's Medical Center, Peking University First Hospital, Beijing, China
Department of Neurology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China
raw_author_name: Hugo C. Moreno
raw_affiliation_strings: Hospital Civil de Guadalajara Fray Antonio Alcalde, Guadalajara, Mexico
raw_author_name: Stacey Kiat Hong Tay
raw_affiliation_strings: Division of Paediatric Neurology, Department of Paediatrics, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore
raw_author_name: Meow-Keong Thong
raw_affiliation_strings: Genetic Medicine Unit, Department of Paediatrics, Faculty of Medicine, University of Malaya, Kuala Lumpur, Malaysia
M. Kandiah Faculty of Medicine and Health Sciences, Universiti Tunku Abdul Rahman, Kajang, Malaysia
raw_author_name: Alfred Peter Born
raw_affiliation_strings: Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark
raw_author_name: Adriana Banzzatto Ortega
raw_affiliation_strings: Hospital Pequeno Príncipe, Curitiba, Brazil
raw_author_name: Yuh-Jyh Jong
raw_affiliation_strings: Department of Biological Science and Technology, National Yang Ming Chiao Tung University, Hsinchu, Taiwan
Departments of Pediatrics and Laboratory Medicine, and Translational Research Center of Neuromuscular Diseases, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan
Graduate Institute of Clinical Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan
raw_author_name: Mohammad A. Al-Muhaizea
raw_affiliation_strings: Neuromuscular Integrated Practice Unit, Neuroscience Centre, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia
raw_author_name: Anna W. Lee
raw_affiliation_strings: Novartis Pharmaceuticals, East Hanover, NJ, USA
raw_author_name: Jeannie Visootsak
raw_affiliation_strings: Novartis Pharmaceuticals, East Hanover, NJ, USA
raw_author_name: Sitra Tauscher-Wisniewski
raw_affiliation_strings: Novartis Gene Therapies, Inc., Bannockburn, IL, USA
raw_author_name: Iulian Alecu
raw_affiliation_strings: Novartis Pharmaceuticals, Basel, Switzerland
raw_author_name: Rutvick Parlikar
raw_affiliation_strings: Novartis Healthcare Pvt. Ltd., Hyderabad, India
raw_author_name: Richard S. Finkel
raw_affiliation_strings: Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA. richard.finkel@stjude.org
raw_author_name: on behalf of the STEER Study Group
raw_author_name: Dũng Chí Vũ
raw_affiliation_strings: Center for Genetics, Molecular Therapy, Metabolism and Endocrinology, Department of Clinical Genetics/Genomics and Molecular Therapy, Vietnam National Children's Hospital, Hanoi, Vietnam
Pediatric Division, Department of Clinical Medicine, Hanoi University of Public Health, Hanoi, Vietnam
raw_author_name: Meow-Keong Thong
raw_affiliation_strings: Genetic Medicine Unit, Department of Paediatrics, Faculty of Medicine, University of Malaya, Kuala Lumpur, Malaysia
M. Kandiah Faculty of Medicine and Health Sciences, Universiti Tunku Abdul Rahman, Kajang, Malaysia
raw_author_name: Adriana Banzzatto Ortega
raw_affiliation_strings: Hospital Pequeno Príncipe, Curitiba, Brazil
raw_author_name: Yuh-Jyh Jong
raw_affiliation_strings: Department of Biological Science and Technology, National Yang Ming Chiao Tung University, Hsinchu, Taiwan
Departments of Pediatrics and Laboratory Medicine, and Translational Research Center of Neuromuscular Diseases, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan
Graduate Institute of Clinical Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan