SANFILIPPO A

Clinical trials for SANFILIPPO A explained in plain language.

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Gene therapy for sanfilippo a: does it last?

Disease control ENROLLING_BY_INVITATION

This study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy in earlier trials. Researchers will monitor safety and how well the therapy controls the disease over time, using tests like the Bayley cognitive scale. No new gene therapy is g…

Matched conditions: SANFILIPPO A

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

Last updated Jun 27, 2026 12:23 UTC

SANFILIPPO A

Get alerted when new SANFILIPPO A trials appear

Gene therapy for sanfilippo a: does it last?