cystic fibrosis

Enterprise Therapeutics‘ experimental compound called ETX001 was able to increase airway fluid secretion and mucus clearance in preclinical models of cystic fibrosis (CF), a study reports. ETX001 is a potentiator of a chloride channel called TMEM16A. Because TMEM16A’s production and function are independent of CFTR — the protein that…

Cystic fibrosis (CF) advocate, author, Olympic torchbearer, and motivational speaker Andy Lipman didn’t get involved in the CF community — even though he himself had the disease — until a friend had him volunteer in a softball charity event.

Proteostasis Therapeutics announced plans to open two Phase 3 clinical trials of its triple combination therapy candidate for cystic fibrosis this year, after a meeting with the chief healthcare regulatory office for the U.K. One of these pivotal trials will be investigating the possibility of organoids being used for personalized…

Tampa General Hospital (TGH) announced that is the first in Florida to use an organ care system that allows donor lungs, hearts, and livers to stay viable and working for hours during transport, and is likely to expand the number of life-saving transplants, including lung transplants for people…

Tiffany Rich is something of a celebrity in the world of cystic fibrosis (CF). Diagnosed at birth, she graduated college while awaiting a double lung transplant. The co-founder of Salty Cysters, she’s been featured in Cosmopolitan magazine, and has written numerous blogs on the disease. Recently, the 28-year-old…

Trikafta is expected to be a “game-changing” therapy for cystic fibrosis (CF), especially for younger patients, say researchers at Children’s Hospital of Richmond at Virginia Commonwealth University (VCU), who were involved…

Cystic fibrosis carriers — individuals who have certain genetic mutations in one of the copies of the CFTR gene — have a significantly increased risk compared with healthy people for several CF-related conditions, a new study shows. The study, “Cystic fibrosis carriers are at increased risk for…

Michael Boyle, MD, has laid out a five-year strategic plan for the Cystic Fibrosis Foundation (CFF), following his Jan. 1 appointment as president and CEO of the Maryland-based charity. In a three-minute video message posted Jan. 9 to the CFF website, Boyle said the 2020-24 plan will focus…

Trikafta, a recently approved treatment for cystic fibrosis (CF), is now available through the U.S. home delivery and specialty pharmacy AllianceRx Walgreens Prime, as part of its limited distribution therapeutic offerings. Trikafta is a next-generation combination of three CF medications: elexacaftor, tezacaftor, and ivacaftor.

Biopharmaceutical company Antabio has been awarded $4.4 million by CARB-X to continue its research for a therapy against Pseudomonas aeruginosa lung infections in people with cystic fibrosis (CF). The funding will be used to support Antabio’s Pseudomonas Elastase Inhibitor (PEi) program, which is seeking to develop…