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Intelligence » Cystic Fibrosis » Last Week

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[News] Tezacaftor–ivacaftor is safe and efficacious in patients with cystic fibrosis with Phe508del mutations

Wednesday, December 13, 2017 -- Two phase 3 double blind, randomised, placebo-controlled trials published in the New England Journal of Medicine show that treatment with the combination of tezacaftor-ivacaftor in patients with cystic fibrosis aged 12 years and older who have the common cystic fibrosis transmembrane conductance regulator (CFTR) gene mutation, Phe508del, is both safe and efficacious.

Proteostasis Therapeutics Announces Positive Clinical Results from Studies of PTI-428 PTI-801 and PTI-808 in Healthy Volunteers and Patients with Cystic Fibrosis

Monday, December 11, 2017 -- CAMBRIDGE, Mass., Dec. 11, 2017 -- Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis...

Protein structure could pave way for effective drugs to treat cystic fibrosis

Thursday, December 14, 2017 -- Biochemists at the University of Zurich have used cryo-electron microscopy to determine the detailed architecture of the chloride channel TMEM16A. This protein is a promising target for the development of effective drugs to treat cystic fibrosis.