Promising new pill for rare childhood brain diseases enters final testing
Disease control
Ongoing
This study tests an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participant…
Phase: PHASE3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC