Amicus Therapeutics
Clinical trials sponsored by Amicus Therapeutics, explained in plain language.
A biotech company developing treatments for rare diseases.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Upcoming readouts
Trials from this sponsor expected to report results within the next 12 months.
- New hope for fabry patients with kidney failure: drug dosing study underway Results expected Dec 2026 (estimated)
- New hope for kids with pompe: enzyme combo trial underway Results expected Mar 2027 (estimated)
- New drug combo hopes to tame rare muscle disease in kids Results expected Jul 2027 (estimated)
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New hope for kids with pompe: enzyme combo trial underway
Disease control OngoingThis study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least…
Phase 3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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New hope for fabry patients with kidney failure: drug dosing study underway
Disease control OngoingThis study tests the safety and how the body processes migalastat in 14 adults with Fabry disease who have severe kidney impairment or are on dialysis. Participants take migalastat capsules, and researchers measure drug levels in blood and dialysate. The goal is to ensure proper …
Phase 3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC