At the 37th European Cystic Fibrosis Society Conference, held June 11-14 in Sweden, representatives from Vertex Pharmaceuticals Inc. presented data from multiple clinical trials evaluating Kalydeco (ivacaftor) in patients with cystic fibrosis due to various mutations. “Not only does KALYDECO lead to significant initial improvements,…
Young people who suffer from physical disabilities or illnesses, such as cystic fibrosis, don’t always receive all of the necessary care and support they need to make a transition to care services. These are the conclusions of a study from the English Care and Quality Commission that reviewed…
For the 27th year, Cystic Fibrosis Research Inc. will hold a national conference to raise awareness of the disease and advocate for continued research and drug development. “Changing the Faces of Cystic Fibrosis: Inspiring Hope” will take place in San Francisco from August 1st through the 3rd, with several presentations designed…
Aptalis Pharma continued to evaluate its pancreatic enzyme products (PEPs) in patients with cystic fibrosis and exocrine pancreatic insufficiency in its Phase 4 clinical trial of Panzytrat® 25,000. Results show that patients taking Panzytrat had better control over their steatorrhea, or fecal fat content. The study was designed to…
After the Food and Drug Administration (FDA) issued rules requiring approval of pancreatic enzyme products (PEPs) in 2004, the cost and availability of the products added another hardship to the lives…
A number of factors influence the health-related quality of life of patients with cystic fibrosis. A new study in the Italian Journal of Pediatrics written by a collaboration of researchers…
Following the recent success of a phase 3b clinical trial for Aztreonam in cystic fibrosis (CF) patients with Burkholderia infections, Gilead Sciences, Inc., a research-based biopharmaceutical company engaged in CF drug discovery and development, completed another phase 3 clinical trial to evaluate the safety of inhalable Aztreonam (AZLI)…
Vertex Pharmaceuticals‘ two-part proof-of-concept study evaluating ivacaftor (under the name KALYDECO) in 24 cystic fibrosis patients shows data consistent with previous preclinical observations. An eight-week open-label period resulted in improved lung function in patients, initiating plans for a future Phase 3 trial. Each of the 24…
A group of thirteen scientists at the University of North Carolina School of Medicine may know why certain cystic fibrosis treatments improve lung function. Their answer: mucins, the proteins within mucus. “Our finding suggests that diluting the concentration of mucins in cystic fibrosis mucus is a key to better…
In some ways, it’s both the best of times and the worst of times for persons afflicted with certain serious disease conditions, such as cystic fibrosis. On the one hand, there have been encouraging and potentially life-changing breakthroughs in terms of therapy options. On the other, these new drugs can…
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