Myozyme 50 mg powder for concentrate for solution for infusion
Sponsors
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Conditions
Pompe diseasePompe disease / glycogenosis type II / acid maltase deficiency
Phase 4
Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease
Not yet recruitingCTIS2024-514255-15-01
Target: 10Updated: 2025-10-20
Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease; Long-term follow-up of patients receiving commercially available Myozyme
Not yet recruitingCTIS2024-518269-92-00
Target: 156Updated: 2024-12-16