Rare Disease Clues Found In Cell's Recycling System

Rare Disease Clues Found In Cell’s Recycling System

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On Monday, United Press International reports that researchers with the National Institutes of Health have uncovered a compound in a cell’s recycling system that could lead to a treatment for a rare genetic disease. NPC1, is a rare, fatal genetic disease that occurs when a faulty gene prevents the removal of cholesterol and other lipids from cells. This causes impaired movement, slurred speech, seizures and dementia. Researchers have developed a drug with the potential to reduce cholesterol and other lipids in cells, to delay the onset of the disease and lessens some of the symptoms.


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