Emily De Los Reyes
Clinical trials sponsored by Emily De Los Reyes, explained in plain language.
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Tracking a hopeful gene therapy for a devastating childhood disease
Disease control OngoingThis study is tracking 10 children with CLN6 Batten disease for up to 5 years after they received a single, experimental gene therapy. The goal is to understand the long-term safety and whether the treatment continues to help slow the progression of this fatal neurological disord…
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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First gene therapy trial targets rare childhood brain disorder
Disease control ENROLLING_BY_INVITATIONThis 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…
Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC