Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey
Author:
Funder
Novartis Gene Therapies, Inc.
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),General Medicine
Link
https://link.springer.com/content/pdf/10.1007/s12325-023-02685-w.pdf
Reference41 articles.
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3. D’Amico A, Mercuri E, Tiziano FD, Bertini E. Spinal muscular atrophy. Orphanet J Rare Dis. 2011;6:71. https://doi.org/10.1186/1750-1172-6-71.
4. Calucho M, Bernal S, Alías L, et al. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases. Neuromuscul Disord. 2018;28(3):208–15. https://doi.org/10.1016/j.nmd.2018.01.003.
5. Finkel R, Bertini E, Muntoni F, Mercuri E. 209th ENMC international workshop: outcome measures and clinical trial readiness in spinal muscular atrophy 7–9 November 2014, Heemskerk, the Netherlands. Neuromuscul Disord. 2015;25(7):593–602. https://doi.org/10.1016/j.nmd.2015.04.009.
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