Margarida Azevedo, MSc,  —

Vertex Pharmaceuticals, a biotech company that discovers, develops, and commercializes medicines for a series of diseases, currently with a clinical development program focused on cystic fibrosis (CF), recently announced its key business priorities for 2016. Vertex currently has two medicines approved for cystic fibrosis treatment: KALYDECO (ivafactor) and ORKAMBI (lumacaftor/ivacaftor).

A new research study may help establish racial equity in cystic fibrosis (CF) newborn screening programs where identification of gene variants in non-white individuals remains suboptimal when compared to the white population. The research paper, “The Spectrum of CFTR Variants in Nonwhite Cystic Fibrosis Patients,” was published in…

In a new study, researchers in the Netherlands demonstrated that children with cystic fibrosis (CF) have a higher risk of developing human rhinovirus infections, and that these last for longer periods of time than in children without the disease. The research article, titled “Frequency and Duration of Rhinovirus Infections in…

A new study report for a first time an “epidemic spread” of Pandoraea pulmonicola, a bacterial species that belongs to the recently identified genus Pandoraea spp. and which is associated with cystic fibrosis (CF), among patients at a CF center in France. The article, published in BMC Infectious Diseases, is titled “Epidemic spread of Pandoraea pulmonicola in a cystic fibrosis…

In a new study, researchers investigated the CFTR gene locus (its location and position in DNA) and the role of elements involved in regulating the chromatin structure and gene expression, discovering a mechanism that coordinates these regulatory elements and maintains normal gene expression levels when structural perturbations occur. The findings…

In a new study, researchers describe the results of a genome-wide screen of Pseudomonas aeruginosa in a yeast, which led to the identification of three potential new Pseudomonas virulence factors. The research paper, entitled “Genome-wide Screen of Pseudomonas aeruginosa in Saccharomyces cerevisiae Identifies New Virulence Factors,” was published in Frontiers in Cellular and…

PTC Therapeutics Inc. has announced the completion of patient enrollment in its second Phase 3 clinical trial of the experimental cystic fibrosis therapy Translarna™. The clinical trial, named ACT CF, is a placebo controlled study designed to evaluate the efficacy of Translarna (ataluren) in patients, age 6 or older, with nonsense…

Concert Pharmaceuticals, Inc., a clinical stage pharmaceutical company that focuses on the development of new small molecule drugs through its DCE (deuterated chemical entity) Platform®, has announced the beginning of Phase 1 multiple ascending dose clinical trial with its cystic fibrosis treatment CTP-656 (deuterium-modified ivacaftor). The announcement follows recent news…

Assistant Professor Sigrid Ladores, PhD, from the School of Nursing, University of Alabama at Birmingham, and doctoral student Leigh Ann Bray are developing a study entitled “Female Fertility in the Age of New CFTR-Modulating Drugs.” The main focus of the research study will be to assess female patients’ understanding of…

OrPro Therapeutics, Inc. has announced it has been granted a patent by the US Patent and Trademark Office (USPTO) for its lead candidate Theradux™, covering its unique treatment ability for cystic fibrosis (CF). The company has been developing a treatment pipeline based on thioredoxin, a naturally secreted protein with enzymatic activity known to have…