Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

BACKGROUND: Spinal muscular atrophy (SMA) is a rare neuromuscular disorder leading to early death in the majority of affected individuals without treatment. Recently, targeted treatment approaches including Onasemnogene Abeparvovec (OA) were introduced. This study describes the first real-world expe...

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Detalles Bibliográficos
Autores principales: Stettner, Georg M., Hasselmann, Oswald, Tscherter, Anne, Galiart, Elea, Jacquier, David, Klein, Andrea
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9971686/
https://www.ncbi.nlm.nih.gov/pubmed/36855136
http://dx.doi.org/10.1186/s12883-023-03133-6

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