News

Proteostasis Therapeutics, Inc., recently presented new and promising early data on its genotype-agnostic amplifier and novel corrector of the cystic fibrosis transmembrane conductance regulator (CFTR), the protein defective in cystic fibrosis (CF). The data, from preclinical studies, were presented in two posters at the 13th Annual European Cystic Fibrosis Society (ECFS)…

When it comes to organ donation, the common scenario is that one patient benefits from another’s misfortune. This time, however, an exceptionally rare heart-lung-heart domino transplant has saved the lives of two lucky women. Diagnosed with cystic fibrosis before the age of one, for Tammy Griffin, receiving both a new…

Early nutritional intervention and monitoring for respiratory and gastrointestinal disease in the youngest cystic fibrosis (CF) patients is vital to improving long-term outcomes. Clinical care guidelines specific to infants with CF, and nutrition and pulmonary guidelines for children over 6 years of age exist, and published by the CF Foundation. However,…

Young children with cystic fibrosis (CF) exhibit a more pro-inflammatory gastrointestinal microbiome, which may underlie impaired nutrient absorption, according to the study “Metagenomic evidence for taxonomic dysbiosis and functional imbalance in the gastrointestinal tracts of children with cystic fibrosis,” published in the journal Scientific…

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 survey that explored the life of patients suffering from cystic fibrosis (CF) pinpointed a number of surprising facts about how patients manage their disease. The survey’s results, while providing some interesting details, are also likely to have implications for research, and identified key points for improvement in clinical practices.

A new technique using relatively inexpensive Microsoft Xbox Kinect gaming devices could soon be used to assess the respiratory health of patients with conditions such as cystic fibrosis (CF). Researchers at the Institute of Digital Healthcare, WMG at the University of Warwick in Coventry, England, the Institute of…

Laurent Pharmaceuticals, Inc., recently announced the completion of a financing round, led by the Cystic Fibrosis Canada, to support a Phase 2 clinical trial of LAU-7b in patients with cystic fibrosis (CF) that is expected to begin this year in the U.S. and Canada. “We are pleased to support Laurent Pharmaceuticals in its effort…

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…