News

Corbus Pharmaceuticals announced that the European Medicines Agency (EMA) has designated the company’s synthetic oral endocannabinoid-mimetic, Resunab (JBT-101), an orphan drug as a potential treatment for people with cystic fibrosis (CF). Resunab, which is currently being evaluated in a Phase 2 clinical trial in CF patients, preferentially binds to the CB2 receptor…

Good Start Genetics  has announced the launch of VeriYou, its new saliva-based screening test for cystic fibrosis (CF) and spinal muscular atrophy (SMA), two common inherited genetic diseases. The screening kit is available for order on the company’s website, for a cost of about $150, and results —…

Researchers at the Perelman School of Medicine at the University of Pennsylvania, Cincinnati Children’s Hospital and Boston University have been awarded a $5.2 million grant, running for seven years, for collaborative work to explore mechanisms of lung regeneration in diseases such as cystic fibrosis. The grant, one…

The U.S. Food and Drug Administration (FDA) approved a new dose of Pertzye (pancrelipase) to specifically treat infants (up to one year of age) with exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) or other conditions. The approval refers to a new capsule containing 4,000 USP units of lipase…

The National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) has granted a five-year, $13.3 million award to Benjamin Gaston, MD, to lead a research program aimed at translating new, small molecule drugs into personalized combination therapies for children with severe asthma and cystic fibrosis (CF). The…

Pulmatrix announced that it will present promising new preclinical data on PUR1900, an investigative inhaled therapy for fungal lung infections in cystic fibrosis patients, in a poster presentation at the North American Cystic Fibrosis Conference (NACFC), taking place Oct. 27–29 in Florida. Patients with cystic fibrosis (CF) often suffer from a condition called allergic bronchopulmonary…

Aradigm Corporation and the Woolcock Institute are collaborating to develop cutting-edge nanotechnologies that target the bacterial and fungal biofilms often present in patients with cystic fibrosis (CF), non-CF bronchiectasis (non-CF BE), and other chronic lung diseases. The most prevalent pathogenic microorganisms found in CF and non-CF BE patients are Haemophilus influenzae, Pseudomonas aeruginosa,…

Researchers at the University of Geneva  discovered that a protein of Pseudomonas aeruginosa, called Host factor q (Hfq), reacts with metals and helps the bacteria gain resistance to multiple classes of antibiotics. P. aeruginosa is an opportunistic bacterial pathogen that causes serious and diverse infections  by producing a broad range of virulence…

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…