New pill aims to tackle rare genetic disorder affecting brain and body
Disease control
Not yet recruiting
This study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…
Phase: PHASE2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC