Eloxx Pharmaceuticals has acquired Zikani Therapeutics and will expand its research program in ribosomal RNA-targeted therapies, with the goal of treating diseases such as cystic fibrosis (CF) caused by a specific type of genetic mutation. …
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Disease progression and the overall health of young people with cystic fibrosis (CF) can be evaluated by putting them through the modified shuttle test (MST) once, as its repeated use showed no significant differences in exercise capacity, a study reports. This test involves repeated movement along…
Thirona announced it has developed a new software program that can quickly detect and quantify lung anomalies in cystic fibrosis (CF) patients, including those beyond the capacity of a doctor in a clinic. The software is based on an artificial intelligence (AI) algorithm, called PRAGMA-AI, that is reported…
The Cystic Fibrosis Foundation (CFF) is awarding Life Edit Therapeutics up to $400,000 to explore its new gene-editing technology to develop treatments for certain patients with cystic fibrosis (CF) who cannot benefit from existing medicines. “We’re looking forward to working with the CF Foundation to leverage the unique…
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…
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