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, Vasantha; Atherton, Mark; Murugan, Archana; Servais, Laurent; Sheehan, Jennie; Standing, Emma; Manzur, Adnan; Scoto, Mariacristina; Baranello, Giovanni; Munot, Pinki; McCullagh, Gary; Willis, Tracey; Tirupathi, Sandya; Horrocks, Iain; Dhawan, Anil; Eyre, Michael; Vanegas, Maria; Fernandez-Garcia, Miguel A.; Wolfe, Amy; Pinches, Laura; Illingworth, Marjorie; Main, Marion; Abbott, Lianne; Smith, Hayley; Milton, Emily; D’Urso, Sarah; Vijayakumar, Kayal; Marco, Silvia Sanchez; Warner, Sinead; Reading, Emily; Douglas, Isobel; Muntoni, Francesco; Ong, Min; Majumdar, Anirban; Hughes, Imelda; Jungbluth, Heinz; Wraige, Elizabeth
Source
In The Lancet Regional Health - Europe February 2024 37
Subject
Language
ISSN
2666-7762