Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease
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چکیده
منابع مشابه
Therapeutic potential of cyclodextrins in the treatment of Niemann-Pick type C disease.
Niemann-Pick type C (NPC) disease is an autosomal recessive neurovisceral lipid and storage disorder characterized by abnormal sequestration of unesterified cholesterol within the late endosomal/lysosomal compartment of all cells in the body. This disease primarily affects children and is characterized by hepatic and pulmonary dysfunction, neurodegeneration and death at an early age. Currently,...
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ژورنال
عنوان ژورنال: Clinical Lipidology
سال: 2012
ISSN: 1758-4299
DOI: 10.2217/clp.12.31