Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disorder that, until recently, was the most common inherited cause of infant mortality. Since 2016, three disease-modifying therapies have emerged, nusinersen, onasemnogene abeparvovec-xioi, and risdiplam, leading to a transformation...

Descripción completa

Detalles Bibliográficos
Autores principales: Ramos-Platt, Leigh, Elman, Lauren, Shieh, Perry B
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2022
Materias:
Perspectives
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9491367/
https://www.ncbi.nlm.nih.gov/pubmed/36157294
http://dx.doi.org/10.2147/IJGM.S369021

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9491367/
https://www.ncbi.nlm.nih.gov/pubmed/36157294
http://dx.doi.org/10.2147/IJGM.S369021

Ejemplares similares