People with a rare genetic form of high cholesterol caused by two broken copies of the LDLR gene don’t get much benefit from PCSK9 inhibitor drugs — most see almost no drop in their bad cholesterol.

People with a rare genetic cholesterol disorder who still have a little bit of working cholesterol-cleaning system in their body tend to respond better to a certain type of cholesterol drug, lowering their bad cholesterol by about 20% on average.

For people with a rare cholesterol condition called HoFH who are already on cholesterol meds, adding a newer drug (PCSK9 inhibitor) barely lowers bad cholesterol for most — and 7 out of 10 don’t get enough benefit to keep taking it.

People with a rare cholesterol condition stayed on a pricey cholesterol drug for about a year on average, even though it didn’t help much — doctors might just keep prescribing it because they’re hesitant to stop treatments that aren’t working.

Even when people with a rare genetic cholesterol disorder take powerful new drugs called PCSK9 inhibitors along with their usual treatments, none of them got their bad cholesterol down to the recommended healthy levels.

People who naturally have a broken PCSK9 gene — which keeps their bad cholesterol low their whole life — are much less likely to get heart disease, and this seems to be true for people of many different backgrounds.