Rare Disease Clues Found In Cell's Recycling System

Rare Disease Clues Found In Cell’s Recycling System

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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