Specific immune cells called macrophages in cystic fibrosis (CF) are unable to kill the bacteria Burkholderia cenocepacia due to impairments in mitochondria, the cell’s powerhouses, a new mouse study says. The finding suggests that boosting mitochondria function could help resolve chronic infection in CF. The study “Defective immunometabolism…
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Children with cystic fibrosis (CF) who become infected with SARS-CoV-2 — the virus that causes COVID-19 — usually have a mild disease course, a new study reports. The findings also indicate that the risk of severe disease may be highest in children with CF who have poorer lung function…
Experimental therapy icenticaftor (QBW251) was able to improve lung function in cystic fibrosis (CF) patients carrying class 3 and 4 mutations in the CFTR gene, results from a Phase 1/2 trial show. It also appeared to be safe and well tolerated when given to both healthy volunteers and CF…
Beyond Air has launched a pilot clinical trial evaluating its investigational therapy LungFit GO — inhaled nitric oxide that can be self-administrated at home — in adults with or without cystic fibrosis (CF) who have chronic, treatment-resistant lung infections caused by non-tuberculous mycobacteria (NTM). Eligible…
Children with cystic fibrosis (CF) who are older, thinner, or have worse lung function may be at increased risk of malnutrition, a study indicates. These findings highlight the importance of screening for malnutrition among children with CF. The study, “Triage for Malnutrition Risk among Pediatric and…
Vertex Pharmaceuticals has awarded a $50,000 grant to Claire’s Place Foundation to support its Work Proudly Program, which helps adults with cystic fibrosis (CF) and caregivers with work-from-home opportunities. “We are extremely grateful for the influx of support for our new Work Proudly Program,” Melissa Yeager,…
BiomX has unveiled a platform designed for more rapid and efficient development of phage therapy, which the company is using to test potential treatments, including one for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). According to a press release, the company expects results of a proof-of-concept…
A clinical trial testing AzurRx BioPharma‘s investigational therapy MS1819, in combination with the current standard care for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), has dosed its first two participants, AzurRx announced. “In the midst of a global pandemic…
The European Commission has approved the combination of Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor) for the treatment of children, ages 6 to 11, with cystic fibrosis (CF) carrying certain genetic mutations. Eligible children must carry either two copies of the F508del mutation — the most common mutation…
Music composed especially for children with cystic fibrosis (CF) made airway clearance therapy a more enjoyable task, suggesting that it can help with treatment adherence and potentially lower costs related to pulmonary exacerbations, a study suggests. The study, “Effects of music therapy as an adjunct to…
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