Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience

BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by a biallelic mutation in the SMN1 gene, resulting in progressive muscle weakness and atrophy. Nusinersen is the first disease-modifying drug for all SMA types. We report on effectiveness and safety data from 120 ad...

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Detalles Bibliográficos
Autores principales: Łusakowska, Anna, Wójcik, Adrianna, Frączek, Anna, Aragon-Gawińska, Karolina, Potulska-Chromik, Anna, Baranowski, Paweł, Nowak, Ryszard, Rosiak, Grzegorz, Milczarek, Krzysztof, Konecki, Dariusz, Gierlak-Wójcicka, Zuzanna, Burlewicz, Małgorzata, Kostera-Pruszczyk, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10401775/
https://www.ncbi.nlm.nih.gov/pubmed/37542300
http://dx.doi.org/10.1186/s13023-023-02769-4

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