SLC6A1

Clinical trials for SLC6A1 explained in plain language.

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First gene therapy trial targets rare childhood brain disorder

Disease control ENROLLING_BY_INVITATION

This is an early-stage study testing a new gene therapy for children with SLC6A1 neurodevelopmental disorder, a rare genetic condition that affects brain development. The therapy involves delivering a working copy of the faulty gene directly into the spinal fluid. The main goal i…

Matched conditions: SLC6A1

Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control

Last updated Mar 30, 2026 14:30 UTC

SLC6A1

Get alerted when new SLC6A1 trials appear

First gene therapy trial targets rare childhood brain disorder