Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Summary

This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to enzyme replacement therapy (ERT) with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first. \- Study visit frequency: every 2 weeks

Timeline

Start

2022-07-11

Primary completion

2026-09-30

Completion

2026-09-30

Drugs

Indications