cystic fibrosis

A clinical trial testing AzurRx BioPharma‘s investigational therapy MS1819, in combination with the current standard care for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), has dosed its first two participants, AzurRx announced. “In the midst of a global pandemic…

The European Commission has approved the combination of Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor) for the treatment of children, ages 6 to 11, with cystic fibrosis (CF) carrying certain genetic mutations. Eligible children must carry either two copies of the F508del mutation — the most common mutation…

Music composed especially for children with cystic fibrosis (CF) made airway clearance therapy a more enjoyable task, suggesting that it can help with treatment adherence and potentially lower costs related to pulmonary exacerbations, a study suggests. The study, “Effects of music therapy as an adjunct to…

The Cystic Fibrosis Foundation (CFF) has awarded up to $3.3 million to Polyphor AG to advance the testing of an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). According to a CFF press…

The Cystic Fibrosis Foundation (CFF) awarded up to $3.75 million to Matinas BioPharma to support preclinical studies of MAT2501, a potential oral antibiotic to treat nontuberculous mycobacterium (NTM) infections in people with cystic fibrosis (CF) and other lung diseases. A goal of this early research work is…

AzurRx BioPharma is requesting a new patient arm be added to its ongoing Phase 2b trial investigating MS1819 capsules as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). The request, filed with the U.S. Food and Drug Administration (FDA), would add a patient group…

Adult age up to 50 and chronic antibiotic use seem to be the greatest risk factors for lung infection by the fungus Aspergillus fumigatus among cystic fibrosis (CF) patients, according to a study in Germany. The study, “Risk factors for respiratory Aspergillus fumigatus in German…

A modified version of Pulmozyme (dornase alfa), a widely-used cystic fibrosis (CF) therapy, lasts longer and reduces the need for treatment from once a day to once a week, a study in mice suggests. The study, “PEGylation of Recombinant Human Deoxyribonuclease I Provides a Long‐Acting Version of…

Following yearlong advocacy efforts, the triple-combination therapy Trikafta is expected to soon come up for approval before Health Canada, the country’s health regulatory agency, the non-profit patient group Cystic Fibrosis Canada has announced on its website. Vertex Pharmaceuticals, which developed and markets Trikafta, its newest modulator therapy for cystic fibrosis and…

New findings from animal studies support the safety and effectiveness of KB407, a potential inhaled gene therapy for cystic fibrosis, its developer, Krystal Biotech, announced. Preclinical work is ongoing, and the therapy is expected to enter clinical testing in patients by mid-2021. “I am particularly encouraged by progress with KB407 for…