Emily De Los Reyes
Clinical trials sponsored by Emily De Los Reyes, explained in plain language.
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Gene therapy for rare brain disease tracked over years
Disease control OngoingThis study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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First gene therapy injection targets rare brain disorder
Disease control ENROLLING_BY_INVITATIONThis early-phase trial tests a one-time gene therapy injection into the spinal fluid for people with SLC6A1 neurodevelopmental disorder, a rare genetic condition. The study involves just 1 participant and will monitor safety and changes in behavior and development over 3 years. T…
Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 26, 2026 17:08 UTC