Margarida Azevedo, MSc,  —

Concert Pharmaceuticals, Inc., which is developing new small molecule drugs through its DCE (deuterated chemical entity) Platform, recently announced positive results from a Phase 1 clinical trial of its cystic fibrosis (CF) treatment, CTP-656 (deuterium-modified ivacaftor). The data, which included a positive safety and tolerability profile, support the continuing development of CTP-656 as…

ACT.md recently reported positive preliminary results from a multiyear project — in partnership with Boston Medical Center and Baystate Health — using the company’s “Collaborative Consultative Care Coordination Program” (4C) to measure improvements in quality of life, family engagement, and healthcare costs for children affected by medically complex conditions like cystic fibrosis. First results…

The European Medicines Authority’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending the designation of orphan medicinal product to Arch Biopartners Inc.‘s AB569 as a potential treatment of infections in cystic fibrosis (CF) patients. AB569 was invented at the University of Cincinnati (UC) in the laboratory…

Pulmatrix, Inc.’s chief scientific officer, Dr. David L. Hava, described in an article recent advances in drug delivery strategies — such as nebulizers for inhaled delivery  — that are helping to treat bacterial and fungal lung infections, and are particularly relevant to people with diseases like cystic fibrosis (CF). The article, “…

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…

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)…

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, “…

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 philanthropic donation to Emory University will contribute to the research of new therapies for cystic fibrosis (CF) at the laboratory of Dr. Eric J. Sorscher, a scientific leader in cystic fibrosis research. Sorscher heads a research program at Emory and Children’s Healthcare in Atlanta that focuses on discovering…