SANFILIPPO A

Clinical trials for SANFILIPPO A explained in plain language.

Never miss a new study

Get alerted when new SANFILIPPO A trials appear

Sign up with your email to follow new studies for SANFILIPPO A, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

Trials to join now! 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 A

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

Last updated Mar 19, 2026 14:56 UTC

SANFILIPPO A

Get alerted when new SANFILIPPO A trials appear

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