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

One-Shot gene therapy aims to halt devastating brain disease in toddlers

Disease control Terminated

This study is testing a one-time gene therapy called RGX-121 for young boys with a severe form of Hunter syndrome that affects the brain. The goal is to see if delivering a working gene directly to the brain can slow or stop the loss of thinking and developmental skills. The stud…

Matched conditions: MPS II

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

Last updated Mar 05, 2026 14:00 UTC

MPS II

Get alerted when new MPS II trials appear

One-Shot gene therapy aims to halt devastating brain disease in toddlers