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
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Published:2024-02
Issue:
Volume:37
Page:100817
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ISSN:2666-7762
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Container-title:The Lancet Regional Health - Europe
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language:en
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Short-container-title:The Lancet Regional Health - Europe
Author:
Gowda VasanthaORCID, Atherton Mark, Murugan Archana, Servais Laurent, Sheehan Jennie, Standing Emma, Manzur Adnan, Scoto Mariacristina, Baranello GiovanniORCID, Munot Pinki, McCullagh Gary, Willis TraceyORCID, Tirupathi Sandya, Horrocks Iain, Dhawan Anil, Eyre Michael, Vanegas MariaORCID, 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 SanchezORCID, Warner Sinead, Reading Emily, Douglas Isobel, Muntoni Francesco, Ong MinORCID, Majumdar Anirban, Hughes Imelda, Jungbluth Heinz, Wraige Elizabeth
Subject
Health Policy,Oncology,Internal Medicine
Reference40 articles.
1. Spinal muscular atrophy;Lunn;Lancet,2008 2. Spinal muscular atrophy (SMA) subtype concordance in siblings: findings from the cure SMA cohort;Jones;J Neuromuscul Dis,2020 3. Spinal muscular atrophy;Prior,1993 4. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review;Verhaart;Orphanet J Rare Dis,2017 5. The survival motor neuron protein in spinal muscular atrophy;Coovert;Hum Mol Genet,1997
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