Sanfilippo syndrome type A

Clinical trials for Sanfilippo syndrome type A explained in plain language.

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Can a direct brain infusion slow sanfilippo syndrome?

Disease control Ongoing

This trial tests an experimental enzyme replacement called GC1130A in children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain. The enzyme is given through a small device placed under the scalp that delivers it directly into the fluid around the bra…

Matched conditions: SANFILIPPO SYNDROME TYPE A

Phase 1 • Sponsor: GC Biopharma Corp • Aim: Disease control

Last updated Sep 09, 2026 17:00 UTC

Sanfilippo syndrome type A

Get alerted when new Sanfilippo syndrome type A trials appear

Can a direct brain infusion slow sanfilippo syndrome?