Amicus Therapeutics

This study tested whether a new two-drug combination (ATB200 given by IV plus AT2221 taken by mouth) is safe and effective for adults with Pompe disease. Researchers enrolled 29 adults, including both those who had previously received standard enzyme replacement therapy and those…

This study followed 16 patients over 12 years old with a specific type of Fabry disease for two years. The goal was to see if the drug migalastat was safe and effective for long-term use in controlling this rare genetic disorder. Researchers tracked side effects, kidney and heart…

This study tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The treatment combines an intravenous drug (ATB200) with an oral pill (AT2221). Researchers compared this new combination to the standard enzyme replacement t…

This study followed 119 adults with late-onset Pompe disease for up to four years to see how safe and effective the treatment ATB200/AT2221 is over the long term. Participants had already completed an earlier study and received regular intravenous infusions plus oral medication. …

This study aimed to understand the full burden of receiving enzyme replacement therapy for Fabry disease, a rare genetic disorder. Researchers observed 82 adult patients and their caregivers across multiple countries to measure the time doctors spent on infusions, the time and tr…