Hope for sanfilippo kids: Brain-Infused drug enters final testing
Disease control
Not yet recruiting
This phase 3 trial tests a drug called tralesinidase alfa in 14 children aged 1-5 with Sanfilippo syndrome type B, a rare genetic disease that causes severe brain damage. The drug is given directly into the brain fluid to replace a missing enzyme. The goal is to see if it can slo…
Phase: PHASE3 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC