A recent study describes a new assay, CFseq, that uses dried blood spots to screen for defects in the gene underlying cystic fibrosis (CF) in newborns, and allows for comprehensive, specific, and rapid early detection of the disease. The study, “Next-Generation Molecular Testing of Newborn Dried Blood Spots for Cystic…
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The U.S. Food and Drug Administration has granted Qualified Infectious Disease Product (QIDP) designation to MP-376, Raptor Pharmaceutical’s inhaled levofloxacin to treat pulmonary infections due to Pseudomonas aeruginosa in patients with cystic fibrosis; non-cystic fibrosis bronchiectasis (BE); and nontuberculous mycobacteria (NTM). Raptor, based in Novato, California, is a global biopharma that’s focused on…
Cystic Fibrosis Foundation leaders and cystic fibrosis (CF) community advocates met at the White House with President Barack Obama, officials from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), and other CF stakeholders last month for the Precision Medicine Initiative (PMI) Summit. The summit was an opportunity…
Volatile compounds released by the bacteria Pseudomonas aeruginosa fuel the growth of a fungal pathogen found in lung infections in cystic fibrosis (CF) patients, according to a study recently published in the American Society for Microbiology (ASM) journal mBio. The study, “Volatile Compounds Emitted by Pseudomonas…
In a new study, researchers at Columbia University Medical Center found that viral infections were associated with pulmonary exacerbations in pediatric patients with cystic fibrosis (CF). They also theorized that viruses might impact the respiratory microbiome, leading to the worsening of clinical outcomes during flares. Results of the study, “…
St. Paul, Minnesota-based RespirTech, specialists in the design and manufacture of Airway Clearance Therapy (ACT) vest devices for persons with lung disorders, recently announced the discovery of a novel way to remotely monitor the treatment of cystic fibrosis (CF) patients. Work leading to the innovations was part…
Nivalis Therapeutics, Inc., recently announced an expanded clinical development plan for N91115, the company’s first-in-class stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in patients with cystic fibrosis (CF). An independent Data Monitoring Committee (DMC) completed a review of interim safety results from the ongoing…
Huge strides have been made in the treatment and management of cystic fibrosis (CF) in the past several decades. According to the U.S. National Institutes of Health (NIH), in 1962 the predicted median survival for cystic fibrosis patients was about 10 years, with few surviving into their teenage years. By the…
The Cystic Fibrosis Foundation (CF Foundation) honored professionals in the Charlotte, North Carolina, area for their business, community leadership, and philanthropy work at its Sixth Annual STANDOUT Awards banquet, with proceeds from the 2015 event going to further the Foundation’s research, care, and educational programs for cystic fibrosis (CF). A banquet highlight was the…
An innovative research center providing tailored physical activity and exercise regimens for young people with cystic fibrosis (CF) has been launched with support for the Cystic Fibrosis Trust. The center will benefit from the skills of an international team of specialists, led by Professor Craig Williams and Dr. Alan Barker from the University of Exeter, U.K.,…
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