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Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond

Of the numerous inherited diseases known to afflict the pediatric population, spinal muscular atrophy (SMA) is among the most common. It has an incidence of approximately one in 10,000 newborns and a carrier frequency of one in 50. Despite its relatively high incidence, SMA remains somewhat obscure...

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Hlavní autoři: Monani, Umrao R, De Vivo, Darryl C
Médium: Artigo
Jazyk:Inglês
Vydáno: 2014
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3955729/
https://ncbi.nlm.nih.gov/pubmed/24648831
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2217/fnl.13.58
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