Fixing A Fatal #GeneticDefect In #Babies With A Bit Of #Genetic #Modification

https://hackaday.com/2025/05/27/fixing-a-fatal-genetic-defect-in-babies-with-a-bit-of-genetic-modification/

Here's a good example of how the US health care system is messed up.

I have a genetic defect. This defect causes hypercholesterolemia. So I have what is called *familial* hypercholesterolemia, because the cause is my genes.

I've been approved for a PCSK9 inhibitor. This is a drug that works only for people who have familial hypercholesterolemia. If you give it to a random guy without my disease, it is going to do nothing to them. (Perhaps hurt them, I'm not sure.)

I've received a letter from my health insurance saying that my approval is expiring soon.

Why?  

This is a genetic defect. A PCSK9 inhibitor is not gene therapy. It does not modify my genetic makeup. That's why I have to keep taking this drug.

Because it is a genetic defect, I'm going to have to be on this drug until I die. Or maybe they are going to find something better, but I expect this "something better" will also require approval because we're wading into highly specialized territory here.

So, again, why ask for approvals every year? It is not like my genetic code is going to fix itself magically.

#PCSK9Inhibitor #USHealthcare #healthcare #approval #hypercholesterolemia #genes #GeneticDefect