Glycogen storage disease type II
Not yet recruitingLong‑term study of Pompe disease in all patients receiving alglucosidase alfa drug combination (miglustat, cipaglucosidase alfa, avalglucosidase alfa)
Long‑term study of Pompe disease in all patients receiving alglucosidase alfa drug combination (miglustat, cipaglucosidase alfa, avalglucosidase alfa)
- Disease:Glycogen storage disease type II
- Substances:Alglucosidase AlfaAvalglucosidase Alfa+2
The Netherlands- Participants:0–17 years · 18–64 years · 65+ years
- Substances:Alglucosidase Alfa
- Sponsor:Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)














