Mini-COMET study: Effects of 97 weeks of avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease who were previously treated with alglucosidase alfaPriya S. Kishnani, S. Grace Prakalapakorn, Yin‐Hsiu Chien et al.|Molecular Genetics and Metabolism|2023Cited by 0
Using a novel measuring device to objectively evaluate the clinical benefit on ptosis in infantile-onset Pompe disease (IOPD): Results from 145 weeks' avalglucosidase alfa dosing in Mini-COMET participants previously treated with alglucosidase alfaPriya S. Kishnani, S. Grace Prakalapakorn, Marguerite C. Weinert et al.|Molecular Genetics and Metabolism|2024Cited by 0