Cystic fibrosis drug
-
I was just watching Boomer Esiason onTv and got curious about his son who has Cystic Fibrosis. I was surprised to learn a med was approved in 2019 that has been remarkably effective at treating the protein deficiency that causes the disease.
https://www.lovewhatmatters.com/cystic-fibrosis-illness-boomer-esiason-chronic-illness/
-
Yeah, Vertex. They’re now working on my disease, also with a small molecule corrector. I know them quite well.
-
They had two molecules wash out but with successively better results. They’re probably 2 years away from clinical trials in their 3rd attempt.
It took them probably 10 years to come up with Trikafta for CF. Though they had successful intermediate products.