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- W2009640755 abstract "Spinal muscular atrophy is caused by homozygous mutation of SMN1. There are several potential approaches to tweaking SMN2 messenger RNA, to raise SMN protein levels to therapeutic levels. One such approach involves small molecules." @default.
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- W2009640755 date "2014-10-30" @default.
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- W2009640755 title "Romancing the Spliceosome to Fight Spinal Muscular Atrophy" @default.
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- W2009640755 doi "https://doi.org/10.1056/nejmcibr1409795" @default.
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