Shire

This study aims to learn about the safety and effects of Replagal, an enzyme replacement therapy, in Indian children and adults with Fabry disease. Participants will receive Replagal infusions every two weeks at a clinic for about one year. The research will monitor side effects …

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…