Amicus Therapeutics

This study tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. The treatment combines an IV drug (ATB200) with a pill (AT2221) to see if it helps patients walk farther and breathe better compared to the curr…

This study followed 119 adults with late-onset Pompe disease who had already completed an earlier trial. They received an intravenous drug (ATB200) plus an oral pill (AT2221) to see if the combination remained safe and effective over the long term. The main focus was on side effe…

This study tested two experimental drugs, ATB200 and AT2221, given together to adults with Pompe disease. The goal was to see if the combination is safe and how the body handles it. 29 participants received the drugs through an IV and a pill. The study found that the treatment wa…

This study looked at the long-term safety and effectiveness of the drug migalastat in 16 people over age 12 with Fabry disease who have certain genetic variants. Fabry disease is a rare genetic disorder that can cause pain, organ damage, and other serious problems. The goal was t…

This study looked at how much time and money it takes for Fabry disease patients and their caregivers to receive enzyme replacement therapy. Researchers watched 82 adults during their regular infusions to measure the work for healthcare providers and the impact on patients' daily…