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Spinal Muscular Atrophy Therapeutics: Where do we Stand?

Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder pathologically characterized by the degeneration of motor neurons in the spinal cord and muscle atrophy. Motor neuron loss often results in severe muscle weakness causing affected infants to die before reaching 2 years of age. Pati...

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Detalles Bibliográficos
Publicado en:Neurotherapeutics
Main Authors: d’Ydewalle, Constantin, Sumner, Charlotte J.
Formato: Artigo
Idioma:Inglês
Publicado: Springer US 2015
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4404440/
https://ncbi.nlm.nih.gov/pubmed/25631888
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13311-015-0337-y
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