Could a diabetes drug help kids with rare PMM2-CDG?
Disease control
Terminated
This study tested an oral drug called epalrestat, originally used for diabetic nerve pain, in children with PMM2-CDG, a rare genetic disorder that causes nerve problems and other symptoms. The trial aimed to see if the drug could safely improve nerve function and metabolic marker…
Phase: PHASE3 • Sponsor: Maggie's Pearl, LLC • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC