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Title
Alkaptonuria
ImagePath
System
amino acid metabolism − tyrosine (homogentisate dioxygenase)
Overview
AR deficiency of homogentisate oxidase → accumulation of homogentisic acid.
Pathophysiology
Pigment deposits in connective tissue (ochronosis); polymer deposition in cartilage & heart valves.
Presentation
Bluish‑black sclerae & ear cartilage, dark urine on standing, arthropathy of large joints in adulthood.
Diagnosis
↑ homogentisic acid in urine/plasma; HGD gene sequencing.
Management
Low‑protein diet restricting Phe & Tyr; nitisinone inhibits upstream pathway; NSAIDs for pain.
Epidemiology
Rare; higher in Slovakia & Dominican Republic.
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