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Niemann-pick Type C1 - Npc1

Rare disease clues found in cell's recycling system. 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. Source 1v.

Niemann-pick Type C1 - Npc1

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