One-Time gene therapy aims to halt sanfilippo syndrome
Cure
Not yet recruiting
Researchers test a one-time gene therapy infusion in children and adults with Sanfilippo syndrome (MPS IIIB), a rare inherited disease that damages the brain and body. The therapy delivers a working copy of the NAGLU gene to help the body make an enzyme that is missing or faulty.…
Phase 1/2 • Sponsor: NeuroGT • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC