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
Resource Type
Article
Authors
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
Primary Research Articles
Language
ISSN
2666-7762