We are a non-profit organization trying to raise awareness and money for research on a cure for Niemann-Pick type C disease. This is a fatal disease with only a drug pill that will only slow down the progression of the disease.|
Details: Niemann-Pick type C disease is an autosomal recessive disorder affecting lipid metabolism (the breakdown and use of fats and cholesterol in the body), in a way which causes harmful amounts of lipids to accumulate in the spleen, liver, lungs, bone marrow, and brain. There is no cure for this disease. The promising drug available to slow the progression of the disease, and improve eye movement, swallowing, and other symptoms is Miglustat, commonly known as Zavesca. Unfortunately, Zavesca is a new drug, and the cost for one 100 mg pill is $100.
Category: Diseases & Conditions