Mini-COMET study: Safety and efficacy data after avalglucosidase alfa dosing for ≥ 145 weeks in participants with infantile-onset Pompe disease (IOPD) who had demonstrated clinical decline or sub-optimal response while receiving alglucosidase alfa
加药
医学
疾病
临床试验
儿科
内科学
作者
David Kronn,Priya S. Kishnani,James Davison,Anaïs Brassier,Alexander Broomfield,Sihoun Hahn,Satoko Kumada,François Labarthe,Hirotaka Ohki,Kristina An Haack,Susan Sparks,Swathi Tammireddy,Atef Zaher,Tianyue Zhou,Yin‐Hsiu Chien