News

The experimental drug Translarna (ataluren) can make the cellular protein-making machinery run the so-called “stop sign” mutation in the cystic fibrosis (CF)-causing CFTR gene, replacing it with amino acids (the protein building blocks) similar enough to allow the protein to do its work. Translarna is currently in Phase 3 clinical…

A team of 12 research partners in seven European countries are engaged in a project to develop a mobile app designed to promote autonomy and help people with cystic fibrosis (CF), their families and caregivers understand the disease and monitor treatment. The researchers are, however, finding that achieving their objective is no…

A preclinical study using animal models of cystic fibrosis (CF) showed that SPX-101, a treatment being developed by Spyryx Biosciences, effectively cleared lung mucus in the animals, regardless of the presence of genetic mutations that promote the development of the disease. Results of this study were presented by John Taylor, Spyryx’s president…

Nitrate intake with food can result in an acute but marked increase in the amount of nitric oxide (NO) being exhaled during respiration, suggests a study conducted by researchers in Ireland. This could be important in cystic fibrosis, which is associated with a decreased level of exhaled NO.

Treating patients with cystic fibrosis (CF) or chronic obstructive pulmonary disease (COPD), infected with the fungus Aspergillus fumigatus, with antifungal therapies may contribute to the adaptation and resistance of this microorganism, and exacerbate lung infection, a new study reported. This finding, in a report by medical researchers in the Netherlands, underscores the…

Vertex Pharmaceuticals reported that the U.S. Food and Drug Administration (FDA) has granted approval for its cystic fibrosis (CF) drug Orkambi to be used to treat children, ages 6 to 11, who have two copies (one inherited from each parent) of the F508del mutation in the CFTR gene. Having two…

Results of a clinical trial evaluating Resunab (JBT-101) in a model of inflammation in healthy subjects indicated that this drug may be a treatment for chronic immune responses linked to tissue inflammation and cystic fibrosis (CF). The results were recently presented at this year’s 6th European Workshop on Lipid Mediators, that…

Pharmaxis announced that Bronchitol (mannitol) has been approved in Russia for the treatment of both pediatric and adult patients with cystic fibrosis (CF), the largest market to date for the medication with this indication. Bronchitol is also approved to treat pediatric and adult CF patients (older than age 6) in Australia, and…

Toxins produced by mold found on nuts and corn can exacerbate cystic fibrosis (CF) and pave the way for airway infections, as they block self-protective mechanisms for the lungs. The study, “Fungal Aflatoxins Reduce Respiratory Mucosal Ciliary Function,” published in the journal Scientific Reports, showed that the toxins…

Proteostasis Therapeutics announced it will present preliminary data from its ongoing Phase 1 clinical trial evaluating ascending doses of PTI-428, an oral treatment for people with cystic fibrosis (CF). The data will be presented at the 30th Annual North American Cystic Fibrosis Conference (NACFC 2016), taking place Oct. 27–29 in Orlando, Florida.