Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Published 2018 View Full Article

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Title
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
Authors
Keywords
-
Journal
JOURNAL OF MEDICINAL CHEMISTRY
Volume 61, Issue 15, Pages 6501-6517
Publisher
American Chemical Society (ACS)
Online
2018-07-26
DOI
10.1021/acs.jmedchem.8b00741

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