The @US_FDA expanded eligibility for @VertexPharma CFTR modulators to >600 ppl w/certain rare #cysticfibrosis mutations. This is BIG NEWS for 2 reasons.
https://news.vrtx.com/press-release/vertex-announces-fda-approvals-trikafta-elexacaftortezacaftorivacaftor-and-ivacaftor (1/5)
1. For those with the rare mutations that are now eligible, these are life-changing meds and this opens up their future! (2/5)
2. This approval was based on in vitro (cell) data, not clinical trial data. This is significant for anyone with a disease or mutation too rare to conduct a full clinical trial (hugely significantly for the rare disease community). (3/5)
Important note: CF nonsense mutations, which are EE's, are not part of this list of newly eligible rare mutations. (4/5)
In addition, many people around the world whose mutations ARE eligible remain unable to access these life-saving meds. Our quest continues until 100% of the CF community has a lifesaving breakthrough--FAST and with nobody left behind. (5/5)
You can follow @EmilysEntourage.
Tip: mention @twtextapp on a Twitter thread with the keyword “unroll” to get a link to it.

Latest Threads Unrolled:
Those who think consciousness raising & movement building are disconnected, or even a distraction from electoral politics have not paid attention to the history of social change.In this thread, we
Read more
The 2020s will be known as the Remote Work decadeA few predictions of what is likely to emerge[ a thread ] Third Space: Office and Working from Home will
Read more
1/29: Have you ever had a concept explained to you that helps frame complex issues you’ve been wrestling with and opens your eyes to new possibilities? A concept that I
Read more
By continuing to use the site, you are consenting to the use of cookies as explained in our Cookie Policy to improve your experience.