Amicus Therapeutics

This program offers early access to an experimental treatment (ATB200/AT2221) for people with Pompe disease who cannot join other clinical trials or choose not to use standard therapy. It aims to provide treatment while collecting safety data. Participants must have a confirmed d…

This study tests a new treatment (cipaglucosidase alfa/miglustat) for children with infantile-onset Pompe disease, a rare genetic disorder that causes muscle weakness and heart problems. The trial includes children from birth to under 18 years old, both those who have and haven't…

This study tests the safety and effectiveness of migalastat in 8 children aged 2 to under 12 with Fabry disease, a rare genetic disorder. The treatment is taken for 12 months. Researchers will monitor side effects and measure how the drug moves through the body. The goal is to se…

This program offers early access to an experimental drug (ATB200/AT2221) for children with infantile-onset Pompe disease who are not getting better with standard treatment. It is for kids up to 18 years old who cannot join other clinical trials. The goal is to provide the drug wh…

This study is a worldwide registry that will follow about 500 people with Pompe disease, including both late-onset and infantile forms. It aims to understand the long-term safety and real-world effectiveness of approved treatments, as well as how the disease progresses without tr…

This study watches 450 adults with Fabry disease to see how well treatments, especially migalastat, work over time. Researchers will measure kidney function and track heart, brain, and kidney events. The goal is to understand long-term safety and quality of life.