Research Kallmann Syndrome Inherited Conditions Epidemiology Huntington's Disease Albinism Autism Genetics Polycystic Kidney Disease Leucism Down Syndrome Cerebral Folate Deficiency ALS Genetic Heterogeneity Inherited Diseases Mitochondrial Genetics Folate Receptor 1 Gene Huntingtin Gene CPS1 Deficiency Ancestry Testing Autism Spectrum Disorder Diagnosis Techniques Pigmentation Disorders Cancer Genetics SPG15 Gene Child Health Hereditary Conditions Disease Progression Duchenne Muscular Dystrophy Research Developments Risk Factors Chromosomal Abnormalities SCN1A Gene SCN1A Mutation Neurological Disorders Neurodegenerative Diseases Congenital Disorders G6PD Deficiency
The antisense therapy boosts SCN1A protein to target the disorder’s genetic cause.
