Medical expenses and care pathways of patients with Pompe receiving myozyme: an observational study based on the French national healthcare database.

ABSTRACT:

Background

Pompe disease is a rare, severe genetic multisystem disorder. It was one of the first genetic muscle diseases to benefit from an innovative therapy-enzyme replacement therapy (ERT)-more than 15 years ago. Despite this progress, data on patient management in community healthcare settings remain scarce, yet essential to understanding the dual economic and clinical burden of the disease. In this context, the French national healthcare claims database offers a valuable resource for comprehensive data collection. To our knowledge, this study is the first to provide a detailed, patient-level analysis of healthcare resource utilization for patients with Pompe disease. Our objective was to estimate the number of patients receiving ERT with alglucosidase alfa (Myozyme®), describe

SUBMITTER: Le Bras A 

PROVIDER: S-EPMC12577294 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature