Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (SMN1) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord leading to skeletal...

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Detalles Bibliográficos
Autores principales: René, Charlotte A., Parks, Robin J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10304132/
https://www.ncbi.nlm.nih.gov/pubmed/37376212
http://dx.doi.org/10.3390/pharmaceutics15061764

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10304132/
https://www.ncbi.nlm.nih.gov/pubmed/37376212
http://dx.doi.org/10.3390/pharmaceutics15061764

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