Author Archives: Ana Pena, PhD

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…

Scientists have created a miniature human pancreas-on-a-chip that may help identify the cause of cystic fibrosis-related diabetes (CFRD), a common and life-threatening complication of cystic fibrosis (CF). Researchers believe the pancreas-on-a-chip will provide insightful information about the biology of CFRD, offering a reliable way to monitor pancreatic cell responses and…

AR-501, an investigational inhaled formulation of gallium citrate to treat chronic lung infections in patients with cystic fibrosis (CF), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The treatment, developed by Aridis Pharmaceuticals, is designed to be given once weekly…

Armata Pharmaceuticals‘ investigational phage therapy AP-PA01 combined with antibiotics holds promise for treating multidrug-resistant Pseudomonas airway infections, updated data from a case study show. A young woman with cystic fibrosis (CF) was successfully treated for a multidrug-resistant Pseudomonas aeruginosa pneumonia using AP-PA01 and antibiotics. At 100 days…

Vertex Pharmaceuticals selected its VX-445 (elexacaftor) triple regimen to apply for regulatory approvals in the U.S. and Europe over the third and fourth quarters of 2019. The therapeutic regimen is intended for the treatment of cystic fibrosis…

A pharmaceutical sciences researcher will use a five-year, $3.3 million grant from the National Institutes of Health (NIH) to develop an inhaled therapy for use by all cystic fibrosis (CF) patients, regardless of the genetic mutation underlying their disease. Researcher Gaurav Sahay, PhD, of Oregon State University (OSU),…

Having to take insulin to treat cystic fibrosis-related diabetes (CFRD) adds to patient distress and sense of a poorer quality of life, a study says, alerting experts to a need to find approaches that might ease this burden. The findings were…

A simple new test may help identify which CFTR modulators are more effective for treating individual patients with cystic fibrosis (CF), early results from a pilot study show. The test finds which compounds are more effective in restoring the…

Using antibiotics to fully eliminate new methicillin-resistant Staphylococcus aureus (MRSA) airway infections appears more effective than waiting for the infection to pass naturally in patients with cystic fibrosis (CF), a study reports. In addition, this approach,…

Bacteria species of the genus Streptococcus and Haemophilus are the most common in airways of children with cystic fibrosis (CF) for their first two years of life, a study reveals. The data suggest these bacteria may play an important role in the early progression of CF, but its clinical significance is still…