We report the case of a girl with Hardikar syndrome who underwent living-donor liver transplantation at 2 years of age. This disease, described in 1992, includes a constellation of abnormalities, such as cleft lip and palate, pigmentary retinopathy, and multiple tubular stenoses (e.g., bile ducts, ureters). Other system involvement is variable. Rotation anomalies of the gut and cardiac abnormalities are frequently present. Pathogenesis remains obscure. Our patient was delivered at 33 weeks of gestation by cesarean section, and was jaundiced, with low birth weight and height. On day 5 after birth, the patient underwent Ladd's surgery for intestinal malrotation. One month later, she developed pyelonephritis and urosepsis. She remained jaundiced and a liver biopsy revealed cirrhosis with regenerating nodules, portal chronic inflammation with bile duct proliferation, and lobular cholestasis. The patient underwent several corrective operations, and at 12 months of age she was diagnosed with Hardikar syndrome. She failed to thrive and had progressive cholestasis and jaundice, coagulation disorders, bilateral ureterostomies, repetitive urinary tract infections, bilateral cleft lip and palate, retinopathy, and gut malrotation. She received a liver transplant at 24 months of age from a living donor. She has had an excellent clinical outcome in liver function without further decline of growth and development.