Amicus Therapeutics

This study aims to check the safety and measure how the drug migalastat behaves in the body of people with Fabry disease who also have severe kidney damage or need dialysis. It will enroll about 14 adults who have a specific genetic type of Fabry disease that responds to this dru…

This study is testing a new combination drug (cipaglucosidase alfa with miglustat) for children with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The main goal is to see if the treatment is safe and how well it works in children from birth up to 18 year…