Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
发现 Risdiplam,一种用于治疗脊髓性肌萎缩症 (SMA) 的选择性运动神经元存活 2 (SMN2) 基因剪接修饰剂
期刊:Journal of Medicinal Chemistry
影响因子:6.8
doi:10.1021/acs.jmedchem.8b00741
Hasane Ratni, Martin Ebeling, John Baird, Stefanie Bendels, Johan Bylund, Karen S Chen, Nora Denk, Zhihua Feng, Luke Green, Melanie Guerard, Philippe Jablonski, Bjoern Jacobsen, Omar Khwaja, Heidemarie Kletzl, Chien-Ping Ko, Stefan Kustermann, Anne Marquet, Friedrich Metzger, Barbara Mueller, Nikola