India, June 30 -- Monopar Therapeutics Inc. (MNPR), a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to ALXN1840 for the treatment of Wilson disease.
Wilson disease is a rare, progressive genetic disorder that impairs the body's ability to eliminate excess copper, resulting in toxic copper accumulation in the liver, brain, and other organs. It is caused by mutations in the ATP7B gene, which plays a key role in maintaining copper homeostasis in the body.
Copper accumulation can lead to a wide range of symptoms, including liver disease as well as psychiatric and neurological symptoms, such as personality changes, tremors, and difficulty walking, s...