Mechanistic convergence and shared therapeutic targets in Niemann‐Pick disease
Niemann‐Pick disease type C (NPC) and Tangier disease are genetically and clinically distinct rare inborn errors of metabolism. NPC is caused by defects in either NPC1 or NPC2; whereas Tangier disease is caused by a defect in ABCA1. Tangier disease is currently without therapy, whereas NPC can be tr...
| Main Authors: | , , , , , , , , , , , , |
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| Format: | Journal article |
| Published: |
Wiley
2019
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