Treatment Frequency Reduction in Pompe Disease

Summary

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.

Timeline

Start

2024-10-01

Primary completion

2026-12-31

Completion

2027-12-31

Drugs

Indications