Shire

This study aims to learn more about the safety and effects of an enzyme replacement therapy called Replagal in Indian children and adults with Fabry disease, a rare genetic disorder. Participants who have not taken Replagal before will receive it as an infusion at a clinic every …

This study is creating a long-term registry to track people with Gaucher disease, a rare genetic condition. It follows patients over time to understand how treatments work in real life and learn about the disease's natural progression. No experimental treatments are given—patient…