Scientific Article Mentions KS

Optimized GENE TARGET Framework Characterizes Barriers to Therapeutic Development for Monogenic Neurodevelopmental Disorders

person Raymond Belanger Deloge et al., Molecular Therapy Advances

calendar_today Oct 15, 2026

Researchers refined GENE TARGET, a tool assessing readiness for genetic therapy development across 57 neurodevelopmental disorders. Major barriers include reliable clinical trial measures, long-term patient data, and early diagnosis. Kleefstra syndrome (EHMT1) scored 32/40, ranking among the top conditions for therapeutic research readiness.