FDA Approves First Treatment for Children With Menkes Disease
United States, Jan. 14 -- The U.S. Food and Drug Administration approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Menkes disease is a neurodegenerative disorder caused by a genetic defect that impairs a child's ability to absorb copper. The disease is characterized by seizures, failure to gain weight and grow, developmental delays, and intellectual disability. It leads to abnormalities of the vascular system, bladder, bowel, bones, muscles, and nervous system. Children with classical Menkes (90% of those with the disease) begin to develop symptoms in infancy and typically do not live past three years. It affects approximately one in every 100,000-250,000 live births worldwide...
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