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- W2775211308 abstract "Spinal muscular atrophy (SMA) type 1—the leading genetic cause of infant mortality—results from an absence of functional copies of the SMN1 gene, which encodes survival motor neuron protein (SMN). The course of this disease might soon change, however, as outlined by two publications on successful trials with SMA therapies. 1 Mendell JR Al-Zaidy S Shell R et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017; 377: 1713-1722 Crossref PubMed Scopus (1215) Google Scholar , 2 Finkel RS Mercuri E Darras BT et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017; 377: 1723-1732 Crossref PubMed Scopus (1152) Google Scholar" @default.
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- W2775211308 date "2018-02-01" @default.
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- W2775211308 title "Genetic therapies for spinal muscular atrophy type 1" @default.
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- W2775211308 doi "https://doi.org/10.1016/s1474-4422(17)30436-2" @default.
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