CYP4V2 and Niemann-Pick disease, type C1: Twelve patients aged at least 2 years (2–39 years of age) with a confirmed diagnosis of NPC1 were randomized to receive one of three IV doses of HP-β-CD (1500 mg/kg, 2000 mg/kg, or 2500 mg/kg) every 2 weeks for 48 weeks.