The Mini-COMET Clinical Trial: Safety and Efficacy of Avalglucosidase Alfa after 97 Weeks of Treatment in Children with Infantile-Onset Pompe Disease Previously Treated with Alglucosidase Alfa
医学
儿科
疾病
内科学
作者
David Kronn,James Davison,Alexander Broomfield,Anaïs Brassier,François Labarthe,Si Houn Hahn,Satoko Kumada,Hirotaka Ohki,S. Grace Prakalapakorn,Catherine Wilson,Kristina An Haack,Olivier Huynh-Ba,Susan Richards,Susan Sparks,Swathi Tammireddy,Tianyue Zhou,Yin‐Hsiu Chien,Priya S. Kishnani