Patricia Inácio, PhD,  science writer—

Concert Pharma recently announced successful results from a Phase 1 single ascending dose clinical trial for their lead investigational drug CTP-656 as a therapy for cystic fibrosis. Cystic fibrosis is a life-threatening disease characterized by a progressive lung function decline and is caused by mutations in the Cftr gene (cystic fibrosis transmembrane…

In a new study entitled “Cysteamine as a future intervention in cystic fibrosis against current and emerging pathogens: a patient-based ex vivo study confirming its antimicrobial and mucoactive potential in sputum,” a team of scientists show for the first time that NovaBiotics’ therapy for Cystic Fibrosis, Cysteamine…

In a new study entitled “Natural Variation in Gene Expression Modulates the Severity of Mutant Phenotypes,” researchers show that disease severity in genetic disorders such as Cystic Fibrosis depends on the genetic background of individuals and not solely on the genetic defect of a particular gene. These…

In a new study entitled “Ultrasound echo-intensity predicts severe pancreatic affection in cystic fibrosis patients,” the authors suggest ultrasonography is a suitable and effective diagnostic tool to evaluate pancreatic function in cystic fibrosis patients. The findings could help physicians better diagnose and treat the pancreatic issues and…

The Committee for Medicinal Products for Human Use (CHMP) issued a positive recommendation for the commercialization of Quinsair (240 mg) as a therapeutic for adult patients with cystic fibrosis with recurrent infections by Pseudomonas aeruginosa. Cystic Fibrosis (CF) is an inherited disease that is characterized by defective mucus secretions…

In a recent study entitled “Modulation of the Maladaptive Stress Response to Manage Diseases of Protein Folding,” a team of researchers showed that in protein misfolding diseases, such as cystic fibrosis and Alzheimer’s disease, the cells’ response to misfolding proteins can actually exacerbate the disease phenotype. The study was published in the …

Cystic Fibrosis (CF) is caused by mutations in the Cftr gene (short for cystic fibrosis transmembrane conductance regulator). The CFTR protein is responsible for transporting chloride and sodium ions in and out of lung and intestinal epithelium cells, and so in the absence of CFTR functional protein, thick mucus…

In a recent study entitled “Subnanometre-resolution electron cryomicroscopy structure of a heterodimeric ABC exporter” published in the online issue of Nature, the authors determined the structure of an ABC transporter protein with a sub nanometer resolution for the first time. The new findings has implications in…

The European Medicines Agency (EMA) recently reviewed products containing the antibiotics colistin or colistimethate sodium (known as polymyxins) and recommended alterations to product sheet information to include safety and the most updated information when applied to treating infections that demonstrated to be resistant to other standard antibiotics. This update…

A new study from Karolinska Institute entitled “Interleukin-26 in Antibacterial Host Defense of Human Lungs: Effects on Neutrophil Mobilization” and published in the American Journal of Respiratory and Critical Care Medicine describes a new mechanism of antibacterial defense in the lungs, mediated by…