MPS iiib (sanfilippo B syndrome)

Clinical trials for MPS iiib (sanfilippo B syndrome) explained in plain language.

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Can a weekly brain infusion slow sanfilippo syndrome?

Disease control Expanded access

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…

Matched conditions: MPS IIIB (SANFILIPPO B SYNDROME)

Sponsor: Spruce Biosciences • Aim: Disease control

Last updated Sep 22, 2026 02:00 UTC

MPS iiib (sanfilippo B syndrome)

Get alerted when new MPS iiib (sanfilippo B syndrome) trials appear

Can a weekly brain infusion slow sanfilippo syndrome?