Synspira Therapeutics’s candidate for treating cystic fibrosis, SNSP113, has received orphan drug designation from the European Medicines’ Agency (EMA) in the European Union. The status was granted by the EMA’s Committee for Orphan Medicinal Products (COMP), which reviews experimental treatments developed for rare diseases. Thick mucus accumulates…
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Bacteria species of the genus Streptococcus and Haemophilus are the most common in airways of children with cystic fibrosis (CF) for their first two years of life, a study reveals. The data suggest these bacteria may play an important role in the early progression of CF, but its clinical significance is still…
An experimental large sugar molecule called poly (acetyl, arginyl) glucosamine, or PAAG, was able to disrupt treatment-resistant biofilms formed by Burkholderia cepacia complex bacteria extracted from patients with cystic fibrosis (CF), according to a study. The study, “In Vitro Activity of a Novel…
Choline supplementation improves lung function and liver health in people with cystic fibrosis (CF), and should be considered as a complementary treatment to current standard care therapies, a study suggests. The findings of the study, “Choline Supplementation in Cystic Fibrosis — The Metabolic and Clinical Impact,”…
Resura Prenatal Test, the first non-invasive prenatal test for single-gene (monogenic) disorders such as cystic fibrosis (CF), has been launched by  biotech company Progenity. The test screens fetal cell-free DNA from a sample of maternal blood. According to the company, this test is the first…
Savara Pharmaceuticals has launched a Phase 2a clinical study to evaluate the effectiveness of Molgradex in cystic fibrosis (CF) patients with chronic nontuberculous mycobacterial (NTM) lung infections. Molgradex is an inhaled form of artificially produced human protein granulocyte-macrophage colony-stimulating factor (GM-CSF), which normally is produced and secreted by…
Pseudomonas aeruginosa, the most common bacteria found in cystic fibrosis (CF) patients, uses a virus to go unnoticed by the host immune system, a new study shows. Treatment with a vaccine designed to help the immune system detect the virus was found to significantly reduce P. aeruginosa’s ability to infect mice,…
Canadian patients with cystic fibrosis (CF) have better respiratory function and higher body mass index (BMI) than French patients, although they have higher blood glucose levels and a greater incidence of CF-related diabetes (CFRD), according to a long-term study. The research was published in an article, “…
Eloxx Pharmaceuticals presented new positive preclinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), demonstrating that the therapy increases the levels of CFTR protein and its correct placement at the cell’s surface in patient-derived organoids. Findings were presented by Shira Landskroner-Eiger, PhD, Eloxx’s principal…
Costs for approved cystic fibrosis (CF) treatments and new approvals — including therapies extended to more age groups or targeting new mutations — could double in price, according to pharmacy benefit manager (PBM) Prime Therapeutics. The cost increases could result in higher premiums for Prime members. According to…
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