Conditions: Pompe Disease
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 113
Healthy volunteers: no

Brief summary:
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

Detailed description:

Arms:
arm 1: Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.

Interventions: intervention 1: Alglucosidase alfa
Intervention descriptions: intervention 1: 4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.

Protocol document: