The European Commission (EC) agreed to expand use of Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to include cystic fibrosis (CF) patients 12 and older who have at least one copy of the CFTR gene with an F508del mutation. The EC approved this combination therapy last year to treat people…
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Blocking an enzyme called phosphodiesterase-4 (PDE4) may be useful in the treatment of dry mouth and cystic fibrosis (CF), new preclinical research suggests. The study, “Inhibition of cAMP-Phosphodiesterase 4 (PDE4) Induces Salivation in Mice,” is being presented virtually at the American Society for Pharmacology and Experimental…
People living with cystic fibrosis (CF) need clear, aggressive, and science-driven COVID-19 vaccine distribution strategies that prioritize their condition, says a coalition of experts. The Global Registry Harmonization Group (GRHG), composed of scientists and patient advocates, including the CF Trust, wrote a letter to the…
Signs of lung disease were evident in most cystic fibrosis (CF) patients with normal lung function in a small study, and related with a higher number of pulmonary exacerbations and longer antibiotic use. These findings, based on a mix of lung health measures, indicate that such disease can precede changes…
A clinical study found that in youth with cystic fibrosis (CF) who are in good physical condition, resistance training favorably regulates heart rate variability (HRV), an indicator of the ability to adjust to stress. HRV, a measure of the variation in…
People with cystic fibrosis (CF) generally are living longer and healthier lives than in the recent past. Now, researchers will conduct a five-year study to look into ways to further increase and improve the lifespan of people with the disease. The idea behind the study comes from a collaborative…
Krystal Biotech is expecting to start a Phase 1 clinical trial by early fall to test KB407Â for cystic fibrosis (CF), after the investigational inhaled gene therapy was found safe and well-tolerated in nonhuman primates. According to Krystal, the positive…
Levels of neutrophil elastase, a pro-inflammatory enzyme normally released by immune cells known as neutrophils, plus a greater diversity and amount of microbes could drive disease severity in cystic fibrosis (CF), a study in two siblings suggests. The study, Two Siblings Homozygous for F508del-CFTR Have Varied…
Routine evaluations of how cystic fibrosis (CF) patients and their families are coping with the burden of therapeutic regimens — usually daily therapies that are complex and time-consuming — are an important tool to enhance treatment adherence and create a strong working relationship between patient and care provider, a…
A bioengineer at Rice University will use a more than $2 million federal grant for a project to “repair” harmful mutations that cause cystic fibrosis (CF) using a potentially more accurate approach to gene editing developed in her lab, the university announced in a press release. The National…
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