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…
Phase: PHASE1, PHASE2 • Sponsor: EveryONE Medicines Inc. • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC