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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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Dettagli Bibliografici
Pubblicato in:Neurotherapeutics
Autori principali: d’Ydewalle, Constantin, Sumner, Charlotte J.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer US 2015
Soggetti:
Accesso online: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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