Genzyme, A Sanofi Company

This study is testing the safety and effectiveness of a drug called avalglucosidase alfa for Chinese children under 18 with infantile-onset Pompe disease, a severe genetic disorder. Participants will receive the drug by IV infusion once every two weeks for one year. The main goal…

This study aims to collect information about pregnancy and infant development for women with Pompe disease, a rare genetic disorder. It will follow women who are pregnant, whether they are receiving standard enzyme replacement therapy or not, and track their babies' growth for th…

This is a global registry that collects health information from people with Gaucher disease to better understand how the disease progresses and how treatments work over time. The study involves 12,000 participants worldwide who are followed through their regular medical care, wit…

This international registry collects health information from 9,000 people with Fabry disease to better understand how this rare genetic condition progresses over time. Participants receive their normal medical care while researchers track their health outcomes, including pregnanc…

This global registry aims to better understand Pompe disease, a rare genetic disorder that causes muscle weakness. It will track 2,000 patients over time, both those receiving treatment and those who are not, to learn how the disease progresses and how treatments work long-term. …