A European Medicines Agency (EMA) committee favors extending the label for Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to treat cystic fibrosis (CF) patients 12 and older who have at least one copy of the CFTR gene with an F508del mutation. The European Commission (EC) approved the same combination last year…
Patient enrollment has been completed for a Phase 2 clinical trial testing the investigational therapy MS1819 in combination with pancreatic enzyme replacement therapy, called PERT, for severe exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). Pancreatic insufficiency, known as EPI, is a complication of CF characterized by…
Translate Bio’s ongoing Phase 1/2 clinical trial investigating MRT5005, its candidate inhalation therapy for cystic fibrosis (CF), found that repeat dosing is generally safe and well-tolerated. MRT5005 is designed to treat CF by delivering messenger RNA (mRNA) with instructions for cells to produce a working CF transmembrane conductance regulator…
Due to persistent bacterial infections, long-chain fatty acids (LCFA) are linked to continued inflammation in cystic fibrosis (CF) patients — and may contribute to pulmonary exacerbations in children with CF, according to a recent study. The scientists said further studies are needed to examine how bacterial functions following antibiotic…
Bronchitol (mannitol), an inhaled mucus-clearing treatment for cystic fibrosis (CF), is now commercially available in the U.S., according to Chiesi USA, which is marketing the therapy in the country. “We are excited to bring the first dry powder inhaled mucoactive agent to the U.S. for adults with CF,…
AzurRx BioPharma announced that its Phase 2b trial evaluating the safety and efficacy of MS1819 in treating exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients is fully enrolled. Thirty adults with CF are taking part in the OPTION 2 study (NCT04375878) comparing MS1819 with the current standard…
Thanks to the Cystic Fibrosis Foundation (CFF), people with cystic fibrosis (CF) in the United States will receive free access to the online wellness and exercise platform Beam through the end of this year. “Wellness plays a critical role in an individual’s quality of life no matter what challenges…
A precise gene-editing tool that reverses single DNA mutations may lead to genetic therapies for people with cystic fibrosis (CF), a study suggests. The study, “Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair…
One need look no further than the name of the Cystic Fibrosis Foundation‘s Impact Grant program to figure out what it does — helping those affected by cystic fibrosis (CF) serve their disease community in creative ways that matter. The CF Foundation is currently accepting applications for the 2021 award…
Molecular analysis of single cells lining the digestive tract found high CFTR activity — the gene associated with cystic fibrosis (CF) — in some cells in the duodenum, or first part, of the small intestine. A comparison of cells lining the stomach of…
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