This study tests a one-time gene therapy (FBX-101) given through an IV to children with Krabbe disease who have already received a stem cell transplant. The goal is to see if it is safe and can improve motor function like walking and coordination. Nine participants will receive t…
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated May 17, 2026 11:21 UTC