News

A 30-year-old woman with cystic fibrosis (CF) and poor lung function gave birth to a healthy child while being treated with Trikafta, reportedly the first such successful CF pregnancy in Switzerland. The mother’s percent predicted forced expiratory volume in one second (ppFEV1) — meaning her expected ability to…

First Wave BioPharma has asked the U.S. Food and Drug Administration (FDA) for permission to start a Phase 2 clinical trial that would test the company’s new formulation of adrulipase in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency. Submitting the application “is an important step…

The Phase 2a clinical trial testing Aridis Pharmaceuticals’ experimental inhaled therapy AR-501 for chronic lung infections in people with cystic fibrosis (CF) is fully enrolled. AR-501, designed to be given once a week, can be self-administered within minutes using a hand-held nebulizer that delivers the medication directly to…

The antibiotic Xenleta (lefamulin) was well tolerated in a Phase 1 clinical trial in adults with cystic fibrosis (CF) and showed a similar pharmacological profile to healthy volunteers. “We are excited to share positive topline results from this important study of Xenleta in patients with CF,” said Christine…

A novel gene-editing tool that allows scientists to “drag-and-drop” sequences of DNA into the genome, without inducing large cuts, could aid in treating genetic diseases like cystic fibrosis (CF), a study reported. The technology, aptly called PASTE (for programmable addition via site-specific targeting elements), “expands the capabilities of genome…

Enrollment of adults at sites across the U.S. is continuing for a Phase 2a trial of AR-501 (gallium citrate), Aridis Pharmaceuticals‘ investigational inhaled therapy for chronic lung infections due to cystic fibrosis (CF). As an inhaled treatment, AR-501 is intended to be self-administered weekly using a hand-held nebulizer that…

A year of insulin treatment improves nutritional status and respiratory function in cystic fibrosis (CF) patients with diabetes, according to a study in Italy. In addition, CF patients treated with insulin at an early stage of abnormal blood sugar levels have reduced hospital admissions due to…

Vertex Pharmaceuticals recently filed an application with the U.S. Food and Drug Administration (FDA) asking that Trikafta, its triple-combination modulator therapy, be approved for children with cystic fibrosis (CF) ages 2 to 5. The company is planning to file similar requests in the European Union and U.K.

The Cold Spring Harbor Laboratory (CSHL) has licensed an artificial intelligence platform, developed by Epistemic AI, to accelerate cystic fibrosis (CF)-related discoveries. The platform, which also contains CF-specific resources, will help the lab’s scientists to accelerate their research by providing easy access to publications and clinical trials,…

The rate of new cases, or incidence of cystic fibrosis (CF) is gradually and annually decreasing in the U.S. and Canada alike — and has been for nearly a quarter of century — a new study found. From 1995 to 2019, CF incidence has dropped at a rate of…