Diagnosis & Disease Information

RNA strands

Cystic Fibrosis Foundation Invests in 2 Experimental Gene Therapies

The Cystic Fibrosis Foundation will invest significantly in 2 investigational gene therapies for patients with cystic fibrosis (CF). The therapies aim to use novel nanoparticle technologies to deliver correct copies of CF transmembrane conductance regulator (CFTR) messenger RNA (mRNA) to lung cells so they can produce functional CFTR proteins. “Messenger RNA therapy has the potential…

Realistic coronavirus cells

Impaired SARS-CoV-2 Viral Entry and Replication Seen in Patients With CF

In patients with cystic fibrosis (CF), destructive CF transmembrane conductance regulator (CFTR) channels alter susceptibility to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, resulting in decreased viral entry and replication in CF cells. The findings were published in Nature Communications. The researchers sought to elucidate whether host factors expressed by the CF epithelia may…

Elexacaftor/Tezacaftor/Ivacaftor Halts Lung Function Decline in Patients With CF

In individuals with cystic fibrosis (CF), treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to halt lung function decline over 2 years, according to a recent analysis published in the Journal of Cystic Fibrosis. The analysis compared participants with CF from 2 phase 3 clinical studies and an open-label extension study with a matched group of…

Exercisers

Multiple Tools Can Help Increase Physical Activity, Aerobic Capacity in Patients With CF

Researchers discovered that the use of wearable technology, text message feedback, and goal setting increased physical activity and aerobic capacity in patients with cystic fibrosis (CF). Their findings were published in the Journal of Cystic Fibrosis. An appropriate exercise program is recommended for all patients with CF because evidence suggests that it reduces lung function…

FDA sign

FDA Clears IND Application for Potential mRNA Therapy for Cystic Fibrosis

The US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for VX-522, an experimental messenger RNA (mRNA) therapy being developed by Vertex Pharmaceuticals and Moderna to treat the underlying cause of cystic fibrosis (CF) in patients who cannot benefit from a CF transmembrane conductance regulator (CFTR) modulator. “Clearance of the…

cystic fibrosis etiology

Novel Pangenotypic Chloride Ion Transporter Shows Promise in Cystic Fibrosis

Preclinical data on TAVT-135, a cell-penetrating peptide conjugate intended to restore chloride ion balance in patients with cystic fibrosis (CF), are promising. Tavanta Therapeutics will present the data at the Cystic Fibrosis Foundation’s North American Cystic Fibrosis Conference, being held November 3-5, 2022, in Philadelphia, Pennsylvania. “While CF transmembrane conductance regulator (CFTR) modulators have revolutionized the…

viral infection bacteria

Clinical Trial on Novel Bacteriophage Therapy for Adults With CF Begins

The Antibacterial Resistance Leadership Group (ARLG), funded by the National Institute of Allergy and Infectious Diseases (NIAID), has launched an early-stage clinical trial to assess the use of a potential bacteriophage (or “phage”) therapy in adults with cystic fibrosis (CF). The trial, announced by the National Institutes of Health, will determine whether the investigational treatment…

Doctor examining female

Patients With CF on Triple Therapy Less Likely to Have Respiratory Failure After COVID-19

Patients with cystic fibrosis (CF) who are being treated with elexacaftor/tezacaftor/ivacaftor have a significantly reduced risk of developing acute respiratory failure after contracting coronavirus disease 2019 (COVID-19), compared with CF patients not on the treatment, a new study published in Cureus found.  Moreover, patients who were not being treated with the triple combination therapy had…

FDA approved

Lumacaftor/Ivacaftor Approved for Children With Cystic Fibrosis Under 2 Years of Age

The US Food and Drug Administration (FDA) has approved the use of lumacaftor/ivacaftor (Orkambi®) in children with cystic fibrosis who are aged 12 to 24 months.  In a press release from Vertex, the developers of lumacaftor/ivacaftor, chief medical officer Carmen Bozic, MD, said that treating patients at an early age is of great importance, as…

exercise equipment

First Consensus Statement on Physical Activity in Patients With CF Released

A novel consensus statement on the recommended physical activity in patients with cystic fibrosis (CF) could aid healthcare workers in guiding their patients toward a better quality of life, according to a study recently published in Chronic Respiratory Disease. “This international, multidisciplinary group has highlighted the important role of [physical activity] and exercise in the…

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