PEDIATRIC SDLT CNS DISORDERS

Clinical trials for PEDIATRIC SDLT CNS DISORDERS explained in plain language.

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Not yet finished but already full! 1

First-of-its-Kind trial offers bespoke genetic medicine for child with devastating brain disease

Disease control Ongoing

This early-phase trial is testing a new, personalized type of medicine for children with rare, severe, and life-threatening brain conditions caused by unique genetic errors. The medicine, called an antisense oligonucleotide (ASO), is custom-designed for a single participant's spe…

Matched conditions: PEDIATRIC SDLT CNS DISORDERS

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

Last updated Mar 23, 2026 15:15 UTC

PEDIATRIC SDLT CNS DISORDERS

Get alerted when new PEDIATRIC SDLT CNS DISORDERS trials appear

First-of-its-Kind trial offers bespoke genetic medicine for child with devastating brain disease