Amicus Therapeutics
Clinical trials sponsored by Amicus Therapeutics, explained in plain language.
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New hope for kids with pompe: enzyme combo trial underway
Disease control OngoingThis study tests a combination of two medicines (cipaglucosidase alfa and miglustat) in children aged 0 to 18 with late-onset Pompe disease, a rare genetic disorder that causes muscle weakness. The goal is to see if the treatment is safe and works better than current enzyme repla…
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 17, 2026 08:48 UTC
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New hope for fabry patients with kidney failure: drug safety study underway
Disease control OngoingThis study looks at the safety and how the body processes the drug migalastat in people with Fabry disease who also have severe kidney disease or are on dialysis. About 14 adults with a specific genetic change that makes them eligible for migalastat will take part. The goal is to…
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:02 UTC