SANFILIPPO SYNDROME

Clinical trials for SANFILIPPO SYNDROME explained in plain language.

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Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1

One-Time gene therapy injection aims to halt devastating childhood disease

Disease control Recruiting now

This study is testing a one-time gene therapy called UX111 for children with Sanfilippo Syndrome Type A, a rare and severe genetic disorder. The therapy involves a single intravenous injection designed to deliver a working copy of a missing gene, with the goal of slowing or stopp…

Matched conditions: SANFILIPPO SYNDROME

Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control

Last updated Mar 19, 2026 14:56 UTC

SANFILIPPO SYNDROME

Get alerted when new SANFILIPPO SYNDROME trials appear

One-Time gene therapy injection aims to halt devastating childhood disease