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

发表于 2026-8-5 13:43:19

期刊：Journal of Medicinal Chemistry

文献作者：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; Nikolai A. Naryshkin; Sergey V. Paushkin; Emmanuel Pinard; Agnès Poirier; Michael Reutlinger; Marla Weetall; Andreas Zeller; Xin Zhao; Lutz Mueller

出版日期：2018-8-9

DOI号：10.1021/acs.jmedchem.8b00741

下载链接：https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.8b00741

文献链接：https://doi.org/10.1021/acs.jmedchem.8b00741

当前文献来源于 American Chemical Society (ACS)。

备注信息：

发表于 2026-8-5 13:43:20

: 当前求助已完成，请重新发起求助。
已完结状态的附件将会在24小时内删除。

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

已采纳

同分类求助

最新回复

站点公告

今日热心研友

（热心研友QQ群）

[ACS] Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA) 一键复制