Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders
小分子抑制糖原合酶 1 用于治疗庞贝病和其他糖原累积症
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.adf1691
Julie C Ullman, Kevin T Mellem, Yannan Xi, Vyas Ramanan, Hanne Merritt, Rebeca Choy, Tarunmeet Gujral, Lyndsay E A Young, Kerrigan Blake, Samnang Tep, Julian R Homburger, Adam O'Regan, Sandya Ganesh, Perryn Wong, Terrence F Satterfield, Baiwei Lin, Eva Situ, Cecile Yu, Bryan Espanol, Richa Sarwaikar