SANFILIPPO SYNDROME

Clinical trials for SANFILIPPO SYNDROME explained in plain language.

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Gene therapy for rare childhood brain disease: tracking kids for years after treatment

Disease control ENROLLING_BY_INVITATION

This study is tracking children with Sanfilippo Syndrome A (MPS IIIA) who received an experimental gene therapy called UX111 in earlier trials. The goal is to monitor their long-term safety and see how well the treatment continues to work over many years. Researchers will check f…

Matched conditions: SANFILIPPO SYNDROME

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

Last updated Mar 23, 2026 15:16 UTC

SANFILIPPO SYNDROME

Get alerted when new SANFILIPPO SYNDROME trials appear

Gene therapy for rare childhood brain disease: tracking kids for years after treatment