New hope for rare childhood brain disease
Disease control
Recruiting now
This early-stage study is testing a new drug called JR-446 in children under 18 with a rare, serious genetic disorder called MPS IIIB (Sanfilippo syndrome type B). The main goals are to check if the drug is safe and to see how it affects the body. Researchers will enroll about 10…
Phase: PHASE1, PHASE2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC