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













