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Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil

Crossref DOI link: https://doi.org/10.1016/j.jped.2024.07.011

Published Print: 2025-01

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

Authors

de Albuquerque, Ana Letícia Amorim https://orcid.org/0009-0006-2978-500X
Chadanowicz, Júlia Kersting https://orcid.org/0000-0002-2362-9202
Bevilacqua, Isabela Possebon https://orcid.org/0000-0002-2008-2336
Staub, Ana Lucia Portella https://orcid.org/0000-0003-3963-1056
Winckler, Pablo Brea https://orcid.org/0000-0003-1952-9222
da Silva, Patricia Zambone https://orcid.org/0000-0002-1923-8997
Fagondes, Simone Chaves https://orcid.org/0000-0001-5173-2488
Ferrari, Renata Salatti https://orcid.org/0000-0001-8992-2607
Trojahn, Claudia Denise de Oliveira https://orcid.org/0009-0008-0729-0254
Sacharuk, Viviane Zechlinski https://orcid.org/0009-0007-0030-6762
Kowalski, Thayne Woycinck https://orcid.org/0000-0001-5799-2272
Donis, Karina Carvalho https://orcid.org/0000-0003-3028-3163
Becker, Michele Michelin https://orcid.org/0009-0008-1232-5896
Saute, Jonas Alex Morales https://orcid.org/0000-0003-1141-6573
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Text and Data Mining valid from 2025-01-01

Text and Data Mining valid from 2025-01-01

Version of Record valid from 2024-10-03
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This article is maintained by: Elsevier

Article Title: Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil

Journal Title: Jornal de Pediatria

CrossRef DOI link to publisher maintained version: https://doi.org/10.1016/j.jped.2024.07.011

Content Type: article

Copyright: © 2024 Sociedade Brasileira de Pediatria. Published by Elsevier Editora Ltda.

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