Cystic fibrosis drug
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wrote on 4 Sept 2021, 00:46 last edited by Mik 9 Apr 2021, 00:46
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/
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wrote on 4 Sept 2021, 01:15 last edited by
Yeah, Vertex. They’re now working on my disease, also with a small molecule corrector. I know them quite well.
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wrote on 4 Sept 2021, 01:42 last edited by
I thought you might. And idea when something might be approved?
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wrote on 4 Sept 2021, 02:13 last edited by jon-nyc 9 Apr 2021, 02:14
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.