Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder resulting in degeneration of α-motor neurons of the anterior horn and proximal muscle weakness. It is the leading cause of genetic mortality in children younger than 2 years. It affects ∼1 in 11,000 live births. In 95% of cases...

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Bibliographische Detailangaben
Hauptverfasser: Cherry, Jonathan J., Kobayashi, Dione T., Lynes, Maureen M., Naryshkin, Nikolai N., Tiziano, Francesco Danilo, Zaworski, Phillip G., Rubin, Lee L., Jarecki, Jill
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2014
Schlagworte:
Review Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4142828/
https://ncbi.nlm.nih.gov/pubmed/25147906
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/adt.2014.587
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