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