New hope for kids with sanfilippo syndrome b?
Disease control
Not yet recruiting
This early-stage trial tests a new drug called JR-446 in 12 children under 6 with MPS IIIB, a rare genetic disorder that damages the brain and body. The main goal is to see if the drug is safe and tolerable. Researchers will also measure changes in certain substances in the body …
Phase: PHASE1, PHASE2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Jul 09, 2026 00:00 UTC