Two cholesterol‑clearing drugs could fill different Niemann-Pick niches

Two Niemann-Pick disease type C therapies built on the same backbone chemistry are racing toward the market, but they are likely to carve up the disease space rather than directly compete.

NPC is caused by mutations in the NPC1 or NPC2 gene, which results in cholesterol accumulation in lysosomes. Cholesterol and other lipids can't move to other parts of the cell, which leads to a lipid buildup in organs, causing damage...