Promising Sugar-Blocking drug trial for rare childhood disease halted early
Disease control
Terminated
This study tested an oral drug called epalrestat in children aged 2 to 17 with PMM2-CDG, a rare genetic disorder that affects sugar processing in the body. The goal was to see if the drug could improve symptoms and lab markers compared to a placebo. The trial was terminated early…
Phase: PHASE3 • Sponsor: Maggie's Pearl, LLC • Aim: Disease control
Last updated May 11, 2026 00:43 UTC