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Arginase Deficiency Therapeutics Market — Gene Therapy and Enzyme Replacement Innovations Redefining a Rare Urea Cycle Disorder
Arginase deficiency — a rare autosomal recessive urea cycle disorder (UCD) in which a genetic deficiency of the arginase-1 enzyme causes toxic accumulation of arginine and ammonia in the blood — affects an estimated 1 in 300,000 to 1,000,000 individuals globally. Despite its rarity, the clinical consequences of untreated arginase deficiency are severe and progressive: intellectual...
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