Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review

ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care. SMA is classified into Types 1...

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Detalhes bibliográficos

Principais autores:	Amin Mehrabian, Peter Auguste, Amy Grove, Anna Brown, Janette Parr, Mubarak Patel, Furqan Butt, Jeremiah Donoghue, Mehdi Yousefi, Jo Parsons
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Wiley 2026-02-01
Colecção:	Annals of Clinical and Translational Neurology
Assuntos:	effectiveness nusinersen risdiplam safety SMA spinal muscular atrophy
Acesso em linha:	https://doi.org/10.1002/acn3.70274
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