학술논문
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
Document Type
Article
Author
Gowda, V.; Sheehan, J.; Standing, E.; Vanegas, M.; Fernandez-Garcia, M.A.; Wolfe, A.; Pinches, L.; Jungbluth, H.; Wraige, E.; Atherton, M.; D'Urso, S.; Ong, M.; Murugan, A.; Smith, H.; Milton, E.; Majumdar, A.; McCullagh, G.; Warner, S.; Reading, E.; Hughes, I.; Manzur, A.; Scoto, M.; Baranello, G.; Munot, P.; Main, M.; Abbott, L.; Muntoni, F.; Servais, L.; Willis, T.; Tirupathi, S.; Douglas, I.; Horrocks, I.; Dhawan, A.; Eyre, M.; Illingworth, M.; Vijayakumar, K.; Marco, S.S.
Source
In: The Lancet Regional Health - Europe . (The Lancet Regional Health - Europe, February 2024, 37)
Subject
Language
English
ISSN
26667762