News

Vertex Pharmaceuticals Incorporated recently announced that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) to its supplemental New Drug Application (sNDA) for Kalydeco (ivacaftor) as a treatment for cystic fibrosis patients ages 2 and older with one of 23 residual function mutations in the cystic fibrosis transmembrane conductance…

Proteostasis Therapeutics, Inc., announced that it recently received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for PTI-428, an investigational oral treatment for cystic fibrosis (CF). CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, resulting in a disruption of ion…

Arch Biopartners, Inc, announced that it recently submitted an orphan drug designation application to the European Medicines Authority (EMA) for its product AB569, a potential treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with cystic fibrosis (CF). The EMA is now reviewing the application, and the Committee for Orphan Medicinal Products…

Three Case Western Reserve University faculty members recently received significant funding from the National Center for Accelerated Innovation (NCAI) to conduct projects and develop novel technologies targeting sickle cell anemia, malaria, and cystic fibrosis (CF). NCAI supports new technologies and innovative products that have been targeted as…

Researchers at Stanford University School of Medicine developed a highly sensitive, specific, rapid and cost-effective assay for cystic fibrosis (CF) screening in newborn babies. The research article detailing the development and performance of the new assay, “Next-Generation Molecular Testing of Newborn Dried Blood Spots for Cystic Fibrosis,” was…

University of Iowa researchers appear to have discovered the long-awaited answer as to why mice that carry cystic fibrosis (CF) mutations do not develop the serious lung disease observed in human CF patients. Their work also revealed a new therapeutic target that might stop CF progression. The study, “Airway acidification initiates host defense abnormalities…

A recent study reported that the presence of transient Group A streptococcus (GAS) in patients with cystic fibrosis (CF) increases the risk of pulmonary exacerbations. The study, “Clinical implications and characterization of Group A Streptoccoccus infections in adults with cystic fibrosis,” was published in the journal…

Parents of a child born with a severe genetic condition such as cystic fibrosis (CF) are greatly affected by their child’s illness and likely to favor early prenatal testing of future pregnancies, researchers at Plymouth University, U.K., report. Their study, “Impact of fetal or child loss on parents’ perceptions of non-invasive prenatal diagnosis for…

CysticLife.org, the largest patient-led social network for the cystic fibrosis (CF) community with more than 8,600 members, has partnered with researchers at the Mayo Clinic to conduct what it says will be the world’s largest CF exercise study. A report by rare disease information…

The Geisel School of Medicine at Dartmouth announced that it has received a $10 million gift from an anonymous donor, plus a $5 million matching donation, also given anonymously, that will be used to accelerate its research into better treatments and, ultimately, a cure for cystic fibrosis (CF). The $15 million in…