Status: Not yet recruiting
Treatment Frequency Reduction in Pompe Disease
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.
Participants needed: 10
Trial details
Phase: Phase 4Age: 50+Biological sex: AllType: InterventionalSponsor: Erasmus Medical CenterUpdated: Aug 28, 2024
Eligibility criteria
LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confi... [+6]
Rapidly progressive muscle weakness [+3]