Maureen Newman,  —

Despite every effort to design a drug effective in helping cystic fibrosis patients with defective CFTR proteins, sometimes drugs are thwarted by mechanisms orchestrated by other proteins within cells. A group of researchers from the Department of Human Genetics at National Health Institute Doutor Ricardo Jorge in Portugal recently published…

Two Phase III clinical trials from Northwestern University’s Feinberg School of Medicine and Stanley Manne Children’s Research Institute at Ann Robert H. Lurie Children’s Hospital of Chicago tested a combination treatment plan in patients with cystic fibrosis. The results show that lumacaftor/ivacaftor, to be marketed under the…

After the Pulmonary-Allergy Drugs Advisory Committee (PADAC) met to discuss the approval of Orkambi, members of the committee voted 12 to 1 recommending Vertex Pharmaceuticals’ new treatment for cystic fibrosis to be approved by the US Food and Drug Administration for marketing. The indication is for cystic…

The Food and Drug Administration (FDA) will be evaluating the new drug application (NDA) for Vertex Pharmaceutical’s Orkambi, indicated to treat cystic fibrosis in patients with the F508del mutation on Tuesday, and will make a final decision for approval/disapproval by July 5. The Pulmonary-Allergy Drugs Advisory Committee (PADAC) will be…

Discovering new compounds to treat cystic fibrosis is now more feasible as a result of work in the collaborative laboratory of Dr. Aleksander Edelman at INSERM in France. The research team identified new compounds using their novel functional assay that tests the function of the CFTR protein following the application…

AffloVest, a wearable, self-contained oscillation vest device from International Biophysics that helps patients with Cystic Fibrosis breathe easier, was previously backed by numerous patient testimonies and is now proven in a new Clinical Study that it increased forced expiratory volume in one second (FEV1) an average…

It is time to celebrate more birthdays. Patients with cystic fibrosis, an orphan disease affecting fewer than 200,000 individuals throughout the world, are experiencing an increase in longevity. Estimates from a study out of Queen’s University Belfast in the United Kingdom predicts a nearly 80% increase by the year 2025…

Researchers are finding ways to better understand infection in cystic fibrosis, enabling research efforts that help discover treatments for patients. Dr. Marvin Whiteley at The University of Texas at Austin created a platform to study Pseudomonas aeruginosa, the most common cause of hospitalization and illness for cystic fibrosis patients. Their work…

One size does not fit all when it comes to cystic fibrosis patients’ symptoms, despite the commonality of a mutated gene. Some patients endure severe life-long airway infections while others are relatively unaffected by their condition. Michael Knowles, MD, professor of pulmonary and critical care medicine at the University…

A phase 2 trial from Savara Pharmaceuticals investigating its lead product AeroVanc to treat methicillin-resistant Staphylococcus aureus (MRSA) infections in cystic fibrosis patients met a key primary endpoint. Patients treated with AeroVanc experienced a significant reduction in MRSA density in their…