Niemann-Pick Disease Type C Treatment; a Medication Called Miglustat (Zavesca) May Be an Option
No effective Niemann-Pick disease type C treatment is available for people with type A or B, but for people with mild to moderate type C, a medication called miglustat (Zavesca) may be an option. However, there is no cure for Niemann-Pick disease. Children usually die from infection or progressive neurological loss. Niemann-Pick is a rare, inherited disease that affects the body's ability to metabolize fat (lipids and cholesterol) within cells. Niemann-Pick disease can affect the bone marrow, spleen, liver, nerves, brain, and, in severe cases, lungs. Niemann-Pick type C is a lysosomal storage disease associated with mutations in NPC1 and NPC2 genes. Niemann-Pick type C (NPC) is a slowly progressive disorder whose principal manifestations are age dependent. In infants, Niemann-Pick disease type C leads to various disorders such as Gaucher disease, idiopathic neonatal cholestasis, alpha-1-antitrypsin deficiency, tyrosinemia, and various congenital infections. According to the Nat...