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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...

詳細記述

保存先:
書誌詳細
出版年:Neurotherapeutics
主要な著者: d’Ydewalle, Constantin, Sumner, Charlotte J.
フォーマット: Artigo
言語:Inglês
出版事項: Springer US 2015
主題:
オンライン・アクセス: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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