cystic fibrosis

Vertex Pharmaceuticals and Arbor Biotechnologies have established a partnership to discover new proteins to advance the development of gene therapies for cystic fibrosis. The companies also plan to develop new genome-editing approaches for four other diseases but haven’t revealed which ones yet. Under the terms of the…

The real estate investment and management firm MC Companies, whose owners are grandparents of children with cystic fibrosis, was recognized for its support of disease research by The Cystic Fibrosis Foundation – Tucson Chapter at its fourth annual Round-Up for a Cure event. Round-Up for a Cure is a yearly fundraising event, put…

After eight years of easing the financial burden of protracted hospital stays, Claire’s Place Foundation has reached the $1 million mark in assistance to families living with cystic fibrosis (CF). The foundation, launched in 2011, provides emotional and financial support to those touched by the disease. To date, its ongoing…

Vertex Pharmaceuticals has announced its intention to submit an application to the Scottish Medicines Consortium (SMC) to review the clinical and cost-effectiveness of the company’s cystic fibrosis (CF) medicines, Orkambi (lumacaftor/ivacaftor), as well as Symkevi (tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor). Following the appraisal of data submitted by…

Throughout the past year, Cystic Fibrosis News Today has brought you daily coverage of important discoveries, treatment developments, clinical trials, and other events related to cystic fibrosis (CF). As we look forward to bringing you more news in 2019, we would like to present you with the 10 most-read stories of 2018.

AzurRx BioPharma announced it has started a Phase 2 clinical trial testing the safety, tolerability, and efficacy of MS1819 Spray Dried (MS1819-SD) therapy in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI). The trial was approved in October by the U.S. Food and Drug Administration. Many…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Researchers tested blood samples from cystic fibrosis (CF) patients and identified distinct gene expression patterns that could lead to patient-tailored treatments, a study reports. The study, “Identification of molecular signatures of cystic fibrosis disease status using plasma-based functional genomics,” was published in Physiological Genomics. To better understand…

Vast Therapeutics announced that it received funding from the Cystic Fibrosis Foundation (CFF) to support further development of BIOC51, the company’s investigative treatment for antibiotic-resistant bacteria, including Pseudomonas aeruginosa, that is often the cause of lung infections in cystic fibrosis (CF) patients. P. aeruginosa infections set in when mucus accumulation…

Purdue University researchers have invented a new way of delivering two antibiotics (colistin and ciprofloxacin) deep into the lungs of cystic fibrosis (CF) patients, enabling much more effective killing of antibiotic-resistant bacteria without exposing patients to high systemic doses of these therapies. “We are providing a promising option to fight…