Dosing & Uses
Niemann–Pick disease Type C
Pending FDA approval for treatment of Niemann-Pick disease Type C
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Pharmacology
Mechanism of Action
Increases the production of cell protective heat shock proteins (HSPs), but only in physiologically stressed cells, such as those impacted by disease; activates proteins that act as pharmacologic chaperones to repair cells and reduce protein misfolding and aggregation, thereby allowing the cells to process accumulated lipid
Patients with Niemann-Pick disease (NPD) type C disease are unable to transport cholesterol and lipids inside of cells, which leads to abnormal accumulation of these substances within various tissues of the body, including brain tissue
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
