PEDIATRIC CNS

Clinical trials for PEDIATRIC CNS explained in plain language.

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Custom Gene-Targeting drug tested in single child with rare brain disease

Disease control Ongoing

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one child with a rare, severe genetic brain disorder. The drug aims to correct the specific genetic mistake causing the disease. The trial focuses on safety and how the drug moves through t…

Matched conditions: PEDIATRIC CNS

Phase: PHASE1, PHASE2 • Sponsor: EveryONE Medicines Inc. • Aim: Disease control

Last updated Jun 27, 2026 11:02 UTC

PEDIATRIC CNS

Get alerted when new PEDIATRIC CNS trials appear

Custom Gene-Targeting drug tested in single child with rare brain disease