MPS II

Clinical trials for MPS II explained in plain language.

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Trials to join now! 2 Not yet finished but already full! 1 Completed 1

Gene therapy trial offers hope for kids with rare brain disease

Disease control Terminated

This study tests a gene therapy called RGX-121 in children with Hunter syndrome, a rare genetic disorder that affects the brain and body. The therapy delivers a working copy of the missing gene to help improve brain development and reduce harmful substance buildup. The trial is f…

Matched conditions: MPS II

Phase: PHASE3 • Sponsor: REGENXBIO Inc. • Aim: Disease control

Last updated May 14, 2026 12:06 UTC

MPS II

Get alerted when new MPS II trials appear

Gene therapy trial offers hope for kids with rare brain disease