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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This past weekend I was honored to deliver the 2019 St. Louis University School of Medicine Pre-Commencement address. My very first pediatric doctor at Cincinnati Children’s Hospital, Robert Wilmott MD, is now the acting dean at the SLU School of Medicine and extended the invite to me not long ago. I so very much appreciate...
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Salt in My Soul is a powerful, heartbreaking and inspiring look at Mallory Smith’s life. There are some spoilers ahead, so use this as a warning. She was a brilliant writer who embodied everything it means to live a shortened life to the absolute fullest. She managed to live independently, hold a job, have a...
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I guess a better question is…. Do you (or does your child) have a nonsense mutation? If the answer is yes – whether it’s a single nonsense mutation or a pair – the Patient Registry over at Emily’s Entourage (EE) is for you! Not to be confused with the Cystic Fibrosis Foundation’s Patient Registry, the...
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I am not someone to speak highly of our federal government, regardless of who is in power, but I don’t think I have ever felt so proud of our elected officials. Yesterday, the Senate Finance Committee, in a bipartisan effort, led by chairman Chuck Grassley (R-Iowa) and ranking member Ron Wyden (D-Oregon) called executives from...
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