The Western Hemisphere’s largest gathering of experts in cystic fibrosis gets underway later this month in Nashville, Tennessee — site of the 33rd annual North American Cystic Fibrosis Conference (NACFC). The Oct. 31–Nov. 2 event, sponsored by the Cystic Fibrosis Foundation (CFF), will attract more than 5,000 delegates…
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Vertex Pharmaceuticals has entered a strategic collaboration with Ribometrix to discover potential RNA-targeted therapies for serious diseases. Vertex is a leader in the market of CFTR modulators for cystic fibrosis (CF), and is the developer of Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combination therapy), three approved CF…
Using a simple shirt — a smart one — is closer to becoming a common method to measure lung function in patients with respiratory conditions, including chronic obstructive pulmonary disease (COPD) and cystic fibrosis. A new study conducted by researchers from Radboud University Medical Center and University of Twente,…
Inhibition of the interleukin-1 receptor — to block thick mucus production and inflammation — is a potential strategy to treat cystic fibrosis (CF) in the early stages of the disease before harmful chronic bacterial infections, a study suggests. The study, “…
Voting for two students to receive two scholarships — valued at $22,000 each — is now open to the public until Oct. 9, pharmaceutical company AbbVie has announced. After the voting period, AbbVie’s 2019 Thriving Undergraduate Scholarship will be awarded to an undergraduate student with CF, and its Thriving…
The investigational therapy MS1819 showed positive safety results in a Phase 2 clinical trial for the treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), AzurRx BioPharma announced. In people with CF, EPI can occur when mucus builds up and blocks the ducts of the…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
Kristen Mudrack, PhD, a chemistry professor at Milligan College in Tennessee, has launched a research initiative to encourage college students to produce and test antibiotics that target and destroy Pseudomonas aeruginosa, a bacteria highly resistant to antibiotics that often causes lung infections in patients with…
The Cystic Fibrosis Foundation (CFF) is giving Eloxx Pharmaceuticals up to $1.61 million to support its planned Phase 2 clinical trial program assessing the safety, tolerability, and chemical properties of ELX-02, Eloxx’s lead investigational compound to treat cystic fibrosis (CF) caused by nonsense, or stop, mutations. The program…
CFTR channel activity is controlled by a molecular process known as phosphorylation. Now, a study suggests that the levels of phosphorylation affect how well Kalydeco works in people with certain mutations in the CFTR gene. The study “VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level” was…
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