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Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes

Crossref DOI link: https://doi.org/10.1016/j.jped.2026.101597

Published Print: 2026-11

Update policy: https://doi.org/10.1016/elsevier_cm_policy

Authors

Santos, Andresa R.B.V. https://orcid.org/0000-0002-8556-7641
Ferreira, Lisiane S. https://orcid.org/0000-0001-7037-1585
Santos, Tamires S. https://orcid.org/0000-0002-5827-9548
Mazzeu, Juliana F. https://orcid.org/0000-0002-6161-0510
Sorte, Ney C.A. Boa https://orcid.org/0000-0001-7068-3319
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Text and Data Mining valid from 2026-11-01

Text and Data Mining valid from 2026-11-01

Version of Record valid from 2026-08-05
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This article is maintained by: Elsevier

Article Title: Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes

Journal Title: Jornal de Pediatria

CrossRef DOI link to publisher maintained version: https://doi.org/10.1016/j.jped.2026.101597

Content Type: article

Copyright: © 2026 The Authors. Published by Elsevier España, S.L.U. on behalf of Sociedade Brasileira de Pediatria.