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"Sphingomyelin/cholesterol" lipidosis
Did you mean "Sphingomyelin/cholesterol" lipidoses?
- ... at least 33 pounds (15 kg) who have Niemann-Pick disease type C (NPC; an inherited disease in which ... to treat the neurologic symptoms in patients with Niemann-Pick disease type C.
- ... 2 years of age and older who have Niemann-Pick disease type C (NPC; an inherited disease in which ... works to treat neurologic symptoms in patients with Niemann-Pick disease type C.