Emerging research reveals critical genetic and molecular underpinnings of neurodevelopmental, immunological, and developmental disorders. AI-enabled drug repurposing identifies FDA-approved vorinostat as a promising candidate for Rett syndrome treatment. Mutations in the IKBKG gene responsible for rare immunodeficiency syndromes are elucidated, advancing clinical understanding. Novel molecular signatures enable stratification of rheumatoid arthritis patients optimizing biologic therapy response. Additionally, immune marker profiling distinguishes subgroups within myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), shedding light on disease heterogeneity and potential tailored interventions.