Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical–Experimental, and Real-World Evidence

Spinal muscular atrophy (SMA) is a rare neuromuscular disease, with an estimated incidence of about 1 in 10,000 live births. To date, three orphan drugs have been approved for the treatment of SMA: nusinersen, onasemnogene abeparvovec, and risdiplam. The aim of this narrative review was to provide a...

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Detalles Bibliográficos
Autores principales: Crisafulli, Salvatore, Boccanegra, Brigida, Vitturi, Giacomo, Trifirò, Gianluca, De Luca, Annamaria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10605203/
https://www.ncbi.nlm.nih.gov/pubmed/37891814
http://dx.doi.org/10.3390/brainsci13101446

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10605203/
https://www.ncbi.nlm.nih.gov/pubmed/37891814
http://dx.doi.org/10.3390/brainsci13101446

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