Neuroscience & Neuroimaging Research Collection

Journal article

Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Abstract:: BACKGROUND: Approved spinal muscular atrophy therapies greatly improve clinical outcomes; however, substantial motor function deficits persist. Apitegromab, a fully human monoclonal antibody, selectively inhibits myostatin activation, improving muscle function. We aimed to assess the safety and efficacy of apitegromab in patients with nonambulatory type 2 or type 3 spinal muscular atrophy receiving nusinersen or risdiplam.

METHODS: SAPPHIRE, a double-b... Expand abstract
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Publication status:: Published

Peer review status:: Peer reviewed

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APA Style

Crawford, T. O., Servais, L., Mercuri, E., Kölbel, H., Kuntz, N., Finkel, R. S., Krueger, J., Batley, K., Young, S. D., Marantz, J. L., Song, G., Yao, B., Zhao, G., Rossello, J., Tirucherai, G. S., Mazzone, E. S., Butterfield, R. J., de la Banda, M. G. G., Seferian, A. M., … Darras, B. T. (2025). Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurology, 24(9), 727–739.

MLA Style

Crawford, TO, et al. “Safety and Efficacy of Apitegromab in Nonambulatory Type 2 or Type 3 Spinal Muscular Atrophy (SAPPHIRE): a Phase 3, Double-Blind, Randomised, Placebo-Controlled Trial.” Lancet Neurology, vol. 24, no. 9, 2025, pp. 727–39.

Chicago Style

Crawford, TO, L Servais, E Mercuri, et al. 2025. “Safety and Efficacy of Apitegromab in Nonambulatory Type 2 or Type 3 Spinal Muscular Atrophy (SAPPHIRE): a Phase 3, Double-Blind, Randomised, Placebo-Controlled Trial.” Lancet Neurology 24 (9): 727–39.
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Publisher copy:: 10.1016/s1474-4422(25)00225-x

Authors

+ Crawford, TO More by this author

Role:: Author

+ Servais, L More by this author

Institution:: University of Oxford
Division:: MSD
Department:: Paediatrics
Oxford college:: Kellogg College
Role:: Author
ORCID:: 0000-0001-9270-4061

+ Mercuri, E More by this author

Role:: Author

+ Kölbel, H More by this author

Role:: Author

+ Kuntz, N More by this author

Role:: Author

More authors...

Contributors

+ SAPPHIRE Study Group

Role:: Contributor

+ Children's Hospital of Philadelphia More from this funder

Funder identifier:: https://ror.org/01z7r7q48

Publisher:: Elsevier
Journal:: Lancet Neurology More from this journal
Volume:: 24
Issue:: 9
Pages:: 727-739
Publication date:: 2025-08-13
Acceptance date:: 2025-06-11
DOI:: 10.1016/s1474-4422(25)00225-x
EISSN:: 1474-4465
ISSN:: 1474-4422
Pmid:: 40818473

Language:: English
Keywords:: pediatric

spinal muscular atrophy

neurodegenerative

clinical research

rare diseases

clinical trials and supportive activities

neurosciences
Pubs id:: 2283147
Local pid:: pubs:2283147
Deposit date:: 2025-11-11
ARK identifier:: ark:/29072/ora_cacbc3901f7943eaa885baf415ec027e

Terms of use

Copyright holder:: Elsevier Ltd.
Copyright date:: 2025
Rights statement:: © 2025 Elsevier Ltd. All rights reserved, including those for text and data mining, AI training, and similar technologies.

Licence:: Terms and Conditions of Use for Oxford University Research Archive

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