According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
Congenital muscular dystrophy (CMD) affected individuals, Kelly Berger and Avery Roberts are making their voices heard. Together they hope to tackle misconceptions surrounding disability and give a loud voice…
Currently, there are an estimated 10,000 known rare diseases (and counting). As we learn more about our genetic makeup, and how genetic alterations can cause disease, we continue to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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