News

Ivacaftor (Kalydeco) treatment improves airway flexibility and capacity in patients with cystic fibrosis (CF), according to the results of a study, “Acute administration of ivacaftor to people with cystic fibrosis and a G551D-CFTR mutation reveals smooth muscle abnormalities,” published in the Journal of Clinical Investigation…

Investors working with Cystic Fibrosis Canada are helping to support a new clinical study by Laurent Pharmaceuticals into its  first-in-class lipid modulator, LAU-7b, targeting the compromised immune-inflammatory response in cystic fibrosis (CF). The financing will be used to prepare a Phase 2 clinical trial of LAU-7b in CF patients in both the United States and…

Over 500 members and volunteers of the Cystic Fibrosis Foundation were present at the recent 2016 Volunteer Leadership Conference, a two-day event honoring those whose efforts have made a difference in the lives of people with cystic fibrosis. Joe O’Donnell was the recipient of the Dream Big Award, in recognition of his…

European researchers reported that a combination of two drugs, cysteamine and epigallocatechin gallate (EGCG), may be used to treat cystic fibrosis (CF) patients with specific mutations. The report, “A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR,“ appeared on April…

Arch Biopartners is collaborating with the University of Cincinnati (UC) for a new drug candidate, AB569, for treating Pseudomonas aeruginosa pulmonary infections in patients with cystic fibrosis. Last year, the U.S. FDA granted orphan drug status to Arch Biopartners’ AB569. The company also applied for orphan drug designation from the…

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…