Amicus Therapeutics

This program provides access to an experimental drug combination (ATB200/AT2221) for children with infantile-onset Pompe disease who are getting worse on their current enzyme replacement therapy. The treatment aims to better control this rare genetic disorder that affects muscles…

This study is testing a new combination treatment (cipaglucosidase alfa with miglustat) for children with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and heart problems. The trial includes both children who have never received enzyme …

This program provides access to an experimental treatment called ATB200/AT2221 for people with Pompe disease who can't join regular clinical trials or whose current enzyme replacement therapy isn't working well enough. It's offered on a case-by-case basis to eligible patients who…

This study aims to understand the long-term effects of the drug migalastat for adults with Fabry disease, a rare genetic disorder. Researchers will follow 450 patients for up to 5 years to see how well the treatment controls the disease, its safety, and its impact on quality of l…

This study is testing a medication called migalastat in children aged 2 to under 12 years old who have Fabry disease, a rare genetic condition. The goal is to see if the drug is safe, how the body processes it, and if it helps control the disease over 12 months of treatment. The …

This study is creating a worldwide registry to collect long-term information from 500 people with Pompe disease. It aims to understand how well current treatments work in real life, monitor their safety over time, and learn about the natural course of the disease in untreated pat…