Research Kallmann Syndrome Huntington's Disease Autism Genetics Epidemiology Inherited Conditions Albinism Leucism Mitochondrial Genetics Autism Spectrum Disorder Huntingtin Gene ALS Genetic Heterogeneity Pigmentation Disorders Inherited Diseases Risk Factors Polycystic Kidney Disease Neurodegenerative Diseases G6PD Deficiency Hereditary Conditions Cancer Genetics CPS1 Deficiency Child Health Down Syndrome Duchenne Muscular Dystrophy Disease Progression SCN1A Mutation Diagnosis Techniques SCN1A Gene Research Developments Ancestry Testing Neurological Disorders Chromosomal Abnormalities SPG15 Gene Congenital Disorders
The antisense therapy boosts SCN1A protein to target the disorder’s genetic cause.