Rare disease clues found in cell's recycling system

Monday, July 17, 2017 - 18:32 in Health & Medicine

Scientists have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular 'recycling' system that helps reduce elevated cholesterol and other accumulated fats in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems.

Read the whole article on Science Daily

More from Science Daily

Latest Science Newsletter

Get the latest and most popular science news articles of the week in your Inbox! It's free!

Check out our next project, Biology.Net