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US08633019B2 Methods for treating spinal muscular atrophy 有权
治疗脊髓性肌萎缩的方法

Methods for treating spinal muscular atrophy
Abstract:
The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
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