Mini-COMET study: Effects of repeat avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfaJames Davison, Xianzhang Meng, S. Grace Prakalapakorn et al.|Molecular Genetics and Metabolism|2021Cited by 1
Mini-COMET study: Individual participant-level responses to treatment in patients with infantile-onset Pompe disease receiving repeated dose regimens of avalglucosidase alfa or alglucosidase alfa who were previously treated with alglucosidase alfaPriya S. Kishnani, David Kronn, Anaïs Brassier et al.|Molecular Genetics and Metabolism|2021Cited by 1
Mini-COMET: Individual-level treatment responses in infantile-onset Pompe disease participants receiving avalglucosidase alfa or alglucosidase alfa who previously received alglucosidase alfaPriya S. Kishnani, David Kronn, Alexander Broomfield et al.|Molecular Genetics and Metabolism|2021Cited by 1
Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical declineDavid Kronn, Priya S. Kishnani, James Davison et al.|Molecular Genetics and Metabolism|2022Cited by 1
Mini-COMET: effects of avalglucosidase alfa on ptosis in participants with infantile-onset Pompe disease previously treated with alglucosidase alfaS. Grace Prakalapakorn, Priya S. Kishnani, James Davison et al.|Molecular Genetics and Metabolism|2021Cited by 0