Marisa Wexler, MS,  senior science writer—

The first participants have been dosed in the first clinical trial testing EDT001, an investigational therapy for cystic fibrosis (CF) being developed by Enterprise Therapeutics. The trial is being conducted in healthy participants (without CF) to assess the investigational medicine’s safety profile. “We are excited to be entering…

The U.S. Food and Drug Administration (FDA) has approved expanding the use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF), ages 6 and older, who have at least one F508del mutation in the CFTR gene or a CFTR mutation that responds to Trikafta in laboratory studies. Use of…

A combination of bacteria-infecting viruses and antibiotics can be used to effectively protect lung cells against infection by Pseudomonas aeruginosa, a bacteria that commonly causes lung infections in people with cystic fibrosis (CF), according to a study led by researchers at the University of Geneva (UNIGE). The model…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

As part of its Path to a Cure initiative, the Cystic Fibrosis Foundation announced three new awards to fund research projects that seek to advance the treatment of cystic fibrosis (CF). The largest award, of up to $2.6 million, was given to Eloxx Pharmaceuticals to aid in the…

Children in the U.S. with cystic fibrosis (CF) tend to have better lung function than young people with the disease in the U.K., a new study has found, with researchers saying the disparity is likely due to differences in treatment between the two countries. In the U.S., children are more…

Vertex Pharmaceuticals has made a $32 million purchase from Concert Pharmaceuticals related to future milestones from the 2017 agreement when Vertex purchased Concert’s investigational treatment for cystic fibrosis (CF), VX-561. According to Roger Tung, PhD, Concert’s president and CEO, the purchase will help Concert as it continues…

Combining AzurRx BioPharma‘s investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in cystic fibrosis (CF) patients with severe exocrine pancreatic insufficiency (EPI), interim data from a Phase 2 clinical trial indicate. “The overarching goal of our MS1819 program is to provide a safe and…

People with cystic fibrosis (CF) experience many barriers that limit their participation in physical activity, ranging from a lack of energy to low motivation to self-consciousness, a recent study highlights. A “tentative but important finding” from the study is that people with CF generally prefer at-home exercise over going…

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…