MPS IIIA

Clinical trials for MPS IIIA explained in plain language.

Never miss a new study

Get alerted when new MPS IIIA trials appear

Sign up with your email to follow new studies for MPS IIIA, 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

Gene therapy for rare brain disease: Long-Term safety check begins

Disease control ENROLLING_BY_INVITATION

This study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy. Researchers will track side effects and measure thinking skills over time to see if the treatment remains safe and effective. The goal is to understand long-term outcomes for t…

Matched conditions: MPS IIIA

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

Last updated May 11, 2026 20:47 UTC

MPS IIIA

Get alerted when new MPS IIIA trials appear

Gene therapy for rare brain disease: Long-Term safety check begins