New hope for kids with rare brain disease: drug trial launches
Disease control
Recruiting now
This study tests a new drug called JR-446 in 10 children under 18 with Sanfilippo B syndrome (MPS IIIB), a rare genetic disease that damages the brain. The main goals are to see if the drug is safe and to measure its effects on the body. Researchers will also check how the drug m…
Phase: PHASE1, PHASE2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated May 26, 2026 12:08 UTC