A Disease Progression Model Comparing Respiratory and Motor Decline in People With Late-Onset Pompe Disease Treated With Cipaglucosidase Alfa Plus Miglustat vs. Alglucosidase Alfa

Pombiliti (cipaglucosidase alfa-atga) - Amicus

Myozyme (alglucosidase alfa) - Sanofi

https://www.ispor.org/conferences-education/conferences/upcoming-conferences/ispor-europe-2025/program/program/paper/ispor-europe-2025/poster-session-1-2/a-disease-progression-model-comparing-respiratory-and-motor-decline-in-people-with-late-onset-pompe-disease-treated-with-cipaglucosidase-alfa-plus-miglustat-vs-alglucosidase-alfa

Amy Dymond, BSc, MSc 1; William Green, BSc, MSc 1; Matthew Crabtree, BSc, MPH 2; Alasdair MacCulloch, BSc, PhD 2; Neil Johnson, BSc 2; Simon Shohet, PhD 3; Vera Gielen, PhD 2; Sophie Clarke, PhD 2; Patrick Deegan, MD 4; Jordi Diaz-Manera, MD, PhD 5.

Nov 11, 2025