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Cure My Disease
Don't miss your cure!
Clinical trials sponsored by Neurogt, explained in plain language.
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