Early-Stop trial tests pill for rare muscle disease
Disease control
Terminated
This study tested the safety and effects of three different dosing schedules of an oral drug called duvoglustat (AT2220) in adults with Pompe disease, a rare genetic disorder that weakens muscles. The goal was to see if the drug was well-tolerated and if it could improve muscle f…
Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Apr 02, 2026 09:12 UTC