And sees firsthand how hard it is. Galvanized by his teenage son’s epilepsy diagnosis, Mayes started a new company to try to shepherd a promising inhaler technology for epilepsy through a key clinical trial

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How an unusual medical case in the 1990s provided a clue for how to treat a fatal muscle disease

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A new paper in the latest issue of Health Affairs is worth a look. “Drugs Cleared Through The FDA’s Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process“

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Stacey Lee, an assistant professor at JHU’s Carey Business School, suggests a more transparent process for patients

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“Only less than 8 percent of enrollees are Hispanic, even though Hispanics comprise 17 percent of the population,” said Dr. Eliseo Pérez-Stable, director of NIH’s National Institute on Minority Health and Health Disparities

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Nolan and Jack Willis and just 10 other boys took part in a clinical trial that led to the approval last fall of the very first drug to treat their rare, deadly muscle disease. Now the Willis boys are again test cases as a different type of medical question comes to the fore: whether insurers will cover the controversial drug, Exondys 51, which can cost more than $1 million a year even though it’s still unclear if it works

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In the summer of 2015, Alexandra Franco got a letter in the mail from a company she had never heard of called AcurianHealth. The letter, addressed to Franco personally, invited her to participate in a study of people with psoriasis, a condition that causes dry, itchy patches on the skin

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Failure to Warn

June 6, 2017

Hundreds died while taking an arthritis drug, but nobody alerted patients. This is the first of two stories by STAT on monitoring the safety of new drugs

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