Expanding therapeutic options for Pompe disease: a new small molecule inhibitor of glycogen synthase 1 (GYS1) shows preclinical promise in Pompe disease.
作者信息
Koch Rebecca L, Cocanougher Benjamin T, Lim Jeong-A, Raben Nina, Kishnani Priya S
机构信息
Division of Medical Genetics, Duke University Medical Center, Durham, NC, USA.
M6P Therapeutics, St. Louis, MO, USA.
出版信息
Ann Transl Med. 2024 Dec 24;12(6):123. doi: 10.21037/atm-24-135. Epub 2024 Nov 15.
Abstract
摘要
Zotero 插件