Can a weekly brain infusion slow sanfilippo syndrome?
Disease control
AVAILABLE
This early-access program tests whether a weekly infusion of tralesinidase alfa directly into the brain's fluid spaces is safe and tolerable for children with Sanfilippo syndrome type B, a rare genetic disorder that causes severe neurological decline. The study enrolls about 10 c…
Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC