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Negulescu's research focuses on the therapy of cystic fibrosis. Cystic fibrosis can be caused by any of the thousands of identified mutations in the CFTR protein, an ion channel that allows chloride ions to pass through. These mutations have been classified into Class I to V, with Class III mutations causing defective channel gates in CFTR despite normal expression of the protein. Via high-throughput screening, his team at Vertex Pharmaceuticals discovered ivacaftor, a small-molecule potentiator that increases the probability that mutated CFTR gates will open. Ivacaftor was approved by the Food and Drug Administration (FDA) for cystic fibrosis patients with one specific Class III mutation in 2012, and has since been approved for mutation classes as well. Of note, the expanded approval in 2017 was based solely on in vitro data, due to the small number of patients carrying those rare mutations making clinical trials impossible.
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Jim Jones, Darin J. Correll, Sandra M. Lechner,Ina Jazic, Xiaopeng Miao, David Shaw, Christopher Simard,Jeremiah D. Osteen, Brian Hare, Alina Beaton, Todd Bertoch,Asokumar Buvanendran,
NEW ENGLAND JOURNAL OF MEDICINEno. 5 (2023): 393-405
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Meredith C Fidler, Alexandra Buckley,James C Sullivan,Marvin Statia,Sylvia F Boj,Robert G J Vries,Anne Munck,Mark Higgins, Matteo Moretto Zita,Paul Negulescu,Fredrick van Goor,Kris De Boeck
Fredrick Van Goor,Sabine Hadida,Peter D J Grootenhuis,Bill Burton, Jeffrey H Stack,Kimberly S Straley,Caroline J Decker,Mark Miller,Jason McCartney,Eric R Olson,Jeffrey J Wine, Ray A Frizzell,
Fredrick Van Goor,Sabine Hadida,Peter D. J. Grootenhuis,Bill Burton,Dong Cao, Tim Neuberger, Amanda Turnbull,Ashvani Singh,John Joubran,Anna Hazlewood,Jinglan Zhou,Jason McCartney,
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