Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15 (vol 7, pg 863, 2023)

Chaplin, Marty ORCID: 0000-0002-7097-8704, Bresnahan, Rebecca ORCID: 0000-0001-5578-1535, Fleeman, Nigel ORCID: 0000-0002-4637-9779, Mahon, James, Houten, Rachel ORCID: 0000-0002-4315-7732, Beale, Sophie, Boland, Angela ORCID: 0000-0002-5435-8644, Dundar, Yenal ORCID: 0000-0002-9847-1622, Marsden, Ashley and Munot, Pinki (2023) Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15 (vol 7, pg 863, 2023) PHARMACOECONOMICS-OPEN, 7 (6). p. 1015. ISSN 2509-4262, 2509-4254

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Abstract

This article was originally published under a CC BY-NC license, but has now been made available under a Creative Commons Attribution 4.0 International License (), which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The PDF and HTML versions of the paper have been modified accordingly. The original version of this article was revised due to a retrospective licence change request.

Item Type:	Article
Uncontrolled Keywords:	32 Biomedical and Clinical Sciences, 3202 Clinical Sciences, Rare Diseases, Spinal Muscular Atrophy, Neurosciences, Pediatric Research Initiative, Neurodegenerative, Musculoskeletal
Divisions:	Faculty of Health & Life Sciences Faculty of Health & Life Sciences > Inst. Population Health Faculty of Health & Life Sciences > Inst. Population Health > Inst. Population Health Faculty of Health & Life Sciences > Inst. Population Health > Health Data Science
Depositing User:	Symplectic Admin
Date Deposited:	13 Nov 2025 09:55
Last Modified:	16 Jun 2026 19:55
DOI:	10.1007/s41669-023-00462-7
Related Websites:	Author Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3195351
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