In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy

Until the recent development of disease-modifying therapeutics, spinal muscular atrophy (SMA) was considered a devastating neuromuscular disease with a poor prognosis for most affected individuals. Symptoms generally present during early childhood and manifest as muscle weakness and progressive para...

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Detalles Bibliográficos
Autores principales: Ojala, Kristine S., Reedich, Emily J., DiDonato, Christine J., Meriney, Stephen D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7915832/
https://www.ncbi.nlm.nih.gov/pubmed/33562482
http://dx.doi.org/10.3390/brainsci11020194

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