Sugar therapy trial targets rare genetic disease
Disease control
Not yet recruiting
This study tests a sugar called D-galactose (AVTX-801) in 8 adults with PGM1-CDG, a rare inherited disorder that affects how the body makes proteins. Participants must already be taking D-galactose. The trial aims to see if the treatment reduces episodes of low blood sugar, liver…
Phase: PHASE2 • Sponsor: Eva Morava-Kozicz • Aim: Disease control
Last updated May 04, 2026 16:18 UTC