What is a Rare Disease?

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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Trikafta is Shipping!

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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Headline About Vertex Drug is Causing a Panic Without Context, I Think We’re Going to Be Okay.

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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