MSR2 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
Abstract
Authors
Amy Dymond William Green Matthew Crabtree Alasdair MacCulloch Neil Johnson Simon Shohet Vera Gielen Sophie Clarke Patrick Deegan Jordi Diaz-Manera