The impact of three SMN2 gene copies on clinical characteristics and effect of disease-modifying treatment in patients with spinal muscular atrophy: a systematic literature review

ObjectiveTo review the clinical characteristics and effect of treatment in patients with spinal muscular atrophy (SMA) and three copies of the SMN2 gene.MethodsWe conducted a literature search in October 2022 to identify English-language clinical research on SMA that included SMN2 copy number accord...

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Bibliographic Details
Main Authors: Claudia Dosi, Riccardo Masson
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-02-01
Series:Frontiers in Neurology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2024.1308296/full