cystic fibrosis

Editor’s note: This is third in a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at potential “next-generation” treatments that range from triple combinations in or readying for clinical trials, to gene editing that has a way to go. The research that led…

Cystic fibrosis patients who failed to show early improvements in lung function while being treated with Orkambi (lumacaftor/ivacaftor) still experienced fewer pulmonary exacerbations than those given a placebo in Phase 3 studies, a pooled analysis of those trials reports. Clinicians should consider the totality of outcomes when examining the…

Mutations in the CFTR gene that associate with severe disease can also lead to cardiac problems — specifically, the development of subclinical left ventricular dysfunction — in cystic fibrosis patients, a study suggests. The study, “Subclinical Left Ventricular Dysfunction is Influenced by Genotype Severity in Patients with Cystic…

The public is invited to vote and help determine this year’s AbbVie Thriving Undergraduate and Thriving Graduate Scholarships among eligible students with cystic fibrosis (CF). The criteria for selecting the winners are academic excellence, creativity, and community involvement/extracurricular activities. Each scholarship totals $25,000 and will be awarded to two deserving students.

Vertex Pharmaceuticals has completed enrollment for two Phase 3 studies testing the triple combination therapy of its new CFTR corrector VX-659 together with tezacaftor (VX-661) and Kalydeco (ivacaftor) in patients with cystic fibrosis (CF). The studies focus on patients with two F508del mutations in the CFTR gene (the…

Editor’s Note: This is the second of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we detail the steps that made three disease-modifying oral treatments for cystic fibrosis possible. Kalydeco (ivacaftor), as Vertex‘s cystic fibrosis potentiator came to be known, was a small…

Beginning Oct. 1, the cystic fibrosis (CF) targeted therapy Orkambi (lumacaftor/ivacaftor) will be included on Australia’s Pharmaceutical Benefits Scheme. The Australian government’s decision resulted from the completion of an agreement with Orkambi’s manufacturer, Vertex Pharmaceuticals, and was supported by the recent positive recommendation from the Pharmaceutical Benefits Advisory Committee…

A research team at The University of Queensland was honored for its work on the spread of bacteria among cystic fibrosis patients, being named a winner of this year’s Australian Museum Eureka Prizes. The prizes recognize excellence in four fields:  research and innovation, science engagement, science leadership, and science education. CF Air, as…

Vertex Pharmaceuticals and Genomics announced a three-year collaboration to better understand genetic variations and their effect on clinical outcomes of diseases with unmet therapeutic needs, including cystic fibrosis. The collaboration also will promote the development of precision medicine, an approach to customize treatment based on each patient’s genetic…

Editor’s note: This is the first of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. We begin by recounting the discoveries that made three disease-modifying treatments for cystic fibrosis — by the same science team — possible in a matter of years.  To the interested…