Dataset from Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency

Menkes disease and occipital horn syndrome (OHS) are forms of copper deficiency that must be diagnosed and treated very early in life to prevent serious developmental problems. However, these and other forms of copper deficiency are not very well understood, and further research is needed to determine whether certain treatments are useful in treating copper deficiency. One such treatment is copper histidinate, a new molecular entity that can be injected directly under the skin to avoid absorption through the gastrointestinal tract. This study investigated the effectiveness, side effects, and dosage of copper histidinate treatment for patients with Menkes, OHS, and unexplained copper deficiency.