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Message: Re: Resverlogix Repays $68.8 Million Loan

The cells in patients with Niemann Pick C have greatly disturbed transport/trafficking of cholesterol inside the cell. The NPC1 and NPC2 proteins are of great importance for the lysosomal/endosomal transport of cholesterol and other lipids. Mainly an issue of metabolizing LDL-derived cholesterol and properly channeling this cholesterol (and other lipids) to other cellular compartments. Results in cellular over-accumulation of cholesterol. Apabetalone wouldn't be expected to circumvent these intracellular cholesterol defects in my opinion. Read the signs and symptoms section of the link you shared and you'll see it has a very large neurological component. Even if these patients do have increased risk of atherosclerosis or chronic kidney disease (I'm not sure that they do), that may be the least of their worries.

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