Study Details
A new drug combination study in children with Infantile-Onset Pompe Disease
(IRB#: IRB_00147223)
The goal of this study is to see if a new drug combination can help children with Pompe Disease. The two drugs in the combination are called cipaglucosidase alfa (ATB200) and miglustat (AT2221). This study will also see how safe this drug combination is.
- All genders
- All Ages
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: All Ages
Volunteers: Volunteers with special conditions
Location: In Person
Will I be paid for my time?
Yes
IRB#: IRB_00147223
PI: Nicola Longo
Department: PEDIATRIC GENETICS
Approval Date: 2023-08-09 06:00:00
Specialties: Pediatric Genetics