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Nature Chemical Biology|June 2, 2015
SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA miceJames Palacino, Susanne E Swalley, Cheng Song, et al.Journal of Medicinal Chemistry|May 24, 2021
Discovery of Icenticaftor (QBW251), a Cystic Fibrosis Transmembrane Conductance Regulator Potentiator with Clinical Efficacy in Cystic Fibrosis and Chronic Obstructive Pulmonary DiseaseDarren Le Grand, Martin Gosling, Urs Baettig, et al.Pageof 2