It’s Rare Disease Week 2020, which ultimately culminates with Rare Disease Day on February 29th (the rarest of days!). In the United States, a rare disease is exactly what it sounds like, a disease that is…well… rare. Specifically, a rare disease is one that affects fewer than 200,000 people. The reason we care so much...
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The 2010’s saw the dawn of the modulators. In a remarkable feat of drug development, four cystic fibrosis-specific medications came to market from the test tube: Kalydeco, Orkambi, Symdeko and Trikafta. (If you want to blow your mind, look at the generic names of the drugs included in the modulators – Ivacaftor, Lumacaftor, Tezacaftor, Elexacaftor...
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Trikafta’s amazing power is…well… amazing, and even more so for children living with cystic fibrosis. Our hope is that when children can start dosing Trikafta, they will be putting off disease progression for decades. That goal is seemingly becoming a reality before our eyes. The cystic fibrosis that I, along with much our adult community,...
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This is mostly a PSA for anyone who may need to hear it, or being told anything else… Trikafta is shipping! I have spent most of my morning watching videos across my social media timelines of people with CF taking their first doses and it’s giving me goosebumps. What a time to be alive! Feel...
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It’s flu shot szn! Time to get out there and do your civic duty (read: don’t be a public health menace). The flu is much more than some sneezing and coughing. It could mean time in the hospital, time away from work and time away from your friends. via GIPHY The flu shot reaches maximum...
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Earlier this week STAT News published a brief article entitled, “Vertex sinks on reports of deaths of patients taking cystic fibrosis drug” without providing too much context. Given the first line of the article was, “Thirty patients have died after taking a Vertex (VRTX) Pharmaceuticals treatment for cystic fibrosis, according to a government database” I...
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We’re getting closer to the finish line! It was announced early this morning that Vertex submitted a New Drug Application to the FDA! Vertex also submitted for a priority review, which, if accepted, “would shorten the FDA’s review of the NDA to eight months from the time of submission, versus a standard review timeline of...
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