News

Results of an online survey, conducted by the cystic fibrosis (CF) community GeneFo, showed that a main concern of CF patients is their intimate lives and sexuality. In an attempt to help CF patients and caregivers to find ways, alone or as a couple, to overcome the physical…

The Italian health insurance system has agreed to cover cystic fibrosis patients’ use of Vertex Pharmaceuticals‘ Orkambi (lumacaftor/ivacaftor). Those eligible for the Italian Medicines Agency coverage are patients 12 and older who have two copies of a particular mutation of the gene that is defective in the disease. That is the…

The U.S. Food and Drug Administration is promoting a targeted treatment approach known as precision medicine to help people with diseases stemming from specific, and often rare, genetic features. Its efforts include expanding the use of approved therapies to other genetic-based conditions, and pushing for the development of more biomarkers…

Synspira’s SNSP113 gives antibiotics more punch against the Burkholderia cepacia infections that cystic fibrosis patients experience, a study reports. The research, published in PLoS One, was titled “Novel glycopolymer sensitizes Burkholderia cepacia complex isolates from cystic fibrosis patients to tobramycin and meropenem.” Lung infections caused by Burkholderia cepacia are difficult to…

A first-grader with cystic fibrosis (CF), Conor Fink, inspired a close family friend to hop on his bicycle and spend his summer pedaling from the West to East Coast — Oregon to New York — to  raise money for Claire’s Place Foundation, a nonprofit helping children and families affected by the disease.

Anthera Pharmaceuticals has received news that could help it accelerate patient enrollment in a Phase 3 clinical trial of Sollpura (liprotamase) as a treatment for a digestive disorder known as cystic fibrosis-triggered exocrine pancreatic insufficiency. The news is that a key Cystic Fibrosis Foundation committee approved the company’s trial design. The sign-off…

A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel. Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared…

A deeper understanding of how bacteria like Pseudomonas aeruginosa acts and survives in people despite antibiotic treatment was the focus of a doctoral thesis, along with a recommendation for antibiotic combinations that might work better those what’s currently in use. The thesis, “Host-pathogen interactions in Pseudomonas aeruginosa invasive and respiratory tract infection,” is by…

A computer program created by physicians in Cincinnati may help cystic fibrosis (CF) patients to keep using those at-home treatments they feel are most effective, by providing them an easy way of sharing their preferences — and a given therapy’s effectiveness — with their doctors. CF patients rely on a variety of home therapies, from…

Proteostasis Therapeutics has filed an Investigational New Drug application with the U.S. Food and Drug Administration for PTI-808 as a treatment for cystic fibrosis. PTI-808 promotes the activity of the cystic fibrosis transmembrane conductance regulator, or CFTR, the protein that is faulty in CF. Scientists call the drug a potentiator…