Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study

Spinal muscular atrophy (SMA) is a genetically inherited recessive neuromuscular disease that causes muscular atrophy and weakness. Onasemnogene abeparvovec (formerly AVXS-101, Zolgensma®, Novartis) is a targeted therapy approved to treat patients with SMA in >40 countries worldwide. This study d...

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Detalles Bibliográficos
Autores principales: Bitetti, Ilaria, Lanzara, Valentina, Margiotta, Giovanna, Varone, Antonio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Brief Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10457192/
https://www.ncbi.nlm.nih.gov/pubmed/35606491
http://dx.doi.org/10.1038/s41434-022-00341-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10457192/
https://www.ncbi.nlm.nih.gov/pubmed/35606491
http://dx.doi.org/10.1038/s41434-022-00341-6

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