Early Copper Histidine Therapy in Menkes Disease

This study evaluated the safety and efficacy of daily subcutaneous Copper Histidine (CuHis) injections given over three years to impact the neurodevelopmental outcomes in infants with Menkes disease. The impact of early (beginning within 1 month of birth corrected for prematurity) versus late CuHis treatment (beginning beyond 1 month of age and after onset of neurological symptoms) was evaluated.