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Personalized medicine, providing treatments tailored to individual factors down to the molecular level, is a promising approach for a highly variable disease like cystic fibrosis (CF). The approach is particularly important for children with CF, a group at particular risk of health complications that last for decades. CF, an inherited disease, is well-positioned to…

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…

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…

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, “…