Amicus Therapeutics

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 tested a new treatment for adults with late-onset Pompe disease, a rare muscle-weakening condition. 125 participants received either the experimental drug combo (ATB200 plus AT2221) or the current standard treatment. Researchers measured how far people could walk in 6 …

This study looked at the long-term safety and effectiveness of a treatment combining two drugs (ATB200 and AT2221) for adults with late-onset Pompe disease, a rare genetic disorder that causes muscle weakness. All 119 participants had already completed an earlier study of the sam…

This study looked at how much time healthcare professionals spend preparing and giving enzyme replacement therapy to adults with Fabry disease, and how the treatment affects patients and their caregivers. Researchers measured tasks like setting up the infusion and checking on pat…