Amicus Therapeutics

This study looks at how the drug migalastat works in people with Fabry disease who also have severe kidney issues or are on dialysis. It involves 14 adults and focuses on safety and how the drug moves through the body. The goal is to make sure the right dose is used for these pat…

This study tests a new treatment (cipaglucosidase alfa plus miglustat) for children aged 0 to 18 with late-onset Pompe disease. It aims to check safety, how the drug moves through the body, and its effects. Participants may have received prior enzyme therapy or be new to treatmen…