cystic fibrosis

Savara Pharmaceuticals is stopping its Phase 2a exploratory clinical study of Molgradex (molgramostim nebulizer solution) to treat nontuberculous mycobacterial (NTM) lung infection in people with cystic fibrosis (CF), the company announced in a press release. The decision to discontinue the open-label ENCORE trial (NCT03597347) stems from an inability to achieve the study’s primary outcome. That goal…

Pregnancy is generally safe for both women with cystic fibrosis (CF) and their child, but those with poorer lung function are at greater risk of having a premature or smaller baby, according to a U.K. population-based study. The study, “The outcome of pregnancy in women with cystic fibrosis:…

A new strategy for detecting very small concentrations of specific molecules could allow for better diagnosis and management of a variety of diseases, including cystic fibrosis (CF). The method was described in the journal Nature Communication by a team from the University of Leeds, U.K., in a study titled…

The U.S. Food and Drug Administration (FDA) is reviewing applications from Vertex Pharmaceuticals to expand the approval for three of the company’s cystic fibrosis (CF) therapies: Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and Kalydeco (ivacaftor). The three novel supplemental new drug applications…

ContraFect will receive funding from the Cystic Fibrosis Foundation to conduct preclinical research about the potential of direct lytic agents, an alternative to conventional antibiotics, to treat lung infections caused by Gram negative antibiotic-resistant bacteria, including Pseudomonas aeruginosa. While antibiotics have had a profound impact on…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

People with cystic fibrosis (CF) in the U.K. have grown more numerous, older, and more diverse, according to the U.K. Cystic Fibrosis Registry’s annual data report for 2019. The report, meant to present a snapshot of both the gains as well as the challenges still faced by…

If Trikafta — Vertex Pharmaceuticals’ next-generation triple combination cystic fibrosis (CF) therapy — were to be available in Canada next year, the median age of CF patients could rise by 9.2 years and the number of deaths drop by 15% over the next 10 years, a forecast study reported.

Proteostasis Therapeutics (PTI) has entered into a definitive merger agreement with Yumanity Therapeutics to leverage their common expertise about protein misfolding to advance the development of disease-modifying therapies for several disorders. The merger should be complete before the end of the year. The combined company will operate under the…

The Cystic Fibrosis Foundation (CFF) has awarded nearly $15 million to 33 organizations that are researching how to improve outcomes for cystic fibrosis (CF) patients who are battling infections. The awards fall under the CFF’s Infection Research Initiative, a large-scale effort to provide $100 million in funding…