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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...

Celý popis

Uloženo v:
Hlavní autoři: Amin Mehrabian, Peter Auguste, Amy Grove, Anna Brown, Janette Parr, Mubarak Patel, Furqan Butt, Jeremiah Donoghue, Mehdi Yousefi, Jo Parsons
Médium: Artigo
Jazyk:Inglês
Vydáno: Wiley 2026-02-01
Edice:Annals of Clinical and Translational Neurology
Témata:
effectiveness
nusinersen
risdiplam
safety
SMA
spinal muscular atrophy
On-line přístup:https://doi.org/10.1002/acn3.70274
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