PEDIATRIC CNS

Clinical trials for PEDIATRIC CNS explained in plain language.

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Not yet finished but already full! 1

Custom gene-targeting drugs offer hope for kids with ultra-rare brain diseases

Disease control Ongoing

This study tests a custom-made genetic medicine called an antisense oligonucleotide (ASO) for one child with a rare, life-threatening brain disorder caused by a unique genetic mistake. The ASO is designed to fix that specific error. The main goal is to check if the treatment is s…

Matched conditions: PEDIATRIC CNS

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

Last updated Apr 23, 2026 12:45 UTC

PEDIATRIC CNS

Get alerted when new PEDIATRIC CNS trials appear

Custom gene-targeting drugs offer hope for kids with ultra-rare brain diseases