Vertex Pharmaceuticals announced that two phase 3 studies involving a combination of tezacaftor / ivacaftor improved the lung function in cystic fibrosis patients that are 12 and older, with the F508del…
Welcome to December 2015 PatientWorthians! 'Tis the season for sharing and do we have some stories for you! As we round #NERVEmber this week, we want to highlight some hope for our…
Happy Monday! This week, we have stories on a potential treatment approach for an aggressive form of mitochondrial disease, an experimental treatment for osteosarcoma earning Rare Pediatric Disease designation, and…
Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…
Happy Monday! This week, we have details on a sad story of a toddler passing away from very rare mesenchymal chondrosarcoma, new research about the genetic risk factors of non-alcoholic…
Happy Friday! This week, we have details on an investigational compound for beta thalassemia. Also, we break down the details of a new study that examines psychiatric illness rates among…
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Novo Nordisk’s higher‑dose formulation of semaglutide, Wegovy HD (7.2 mg once weekly), for chronic weight management. The…
GSK has announced a significant advancement in liver disease treatment with FDA approval of Lynavoy (linerixibat), marking the first medication specifically approved in the United States for treating cholestatic pruritus…
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