Vertex Pharmaceuticals Incorporated recently received approval from the United States Food and Drug Administration for Kalydeco® (ivacaftor) to treat children aged between 2 and 5 who suffer with cystic fibrosis (CF) and carry specific mutations in the CFTR gene. Children who carry one of ten different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, including G551D,…
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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…
Research and Markets recently released their “Cystic Fibrosis Market in Europe 2015-2019“ report, which offers an overview of current activities involving pipeline therapeutics in the ongoing fight against cystic fibrosis. Cystic fibrosis, also known as mucoviscidosis, is a genetically inherited disease. Its primary symptoms include breathing difficulties, elevated…
The U.S. Food and Drug Administration (FDA) has classified Kitabis Pak in its Orange Book (an FDA resource listing approved drug products with therapeutic equivalence evaluations) as a therapeutic equivalent to TOBI (tobramycin inhalation solution, USP) for cystic fibrosis (CF). The administration granted it an “AN…
Concert Pharmaceuticals Inc. recently announced that their Phase 1 clinical program for deuterium-modified ivacaftor, a potentially disease-modifying treatment to address cystic fibrosis, has been initiated. Ivacaftor is available under the name Kalydeco®. This Phase 1 trial will be a crossover assessment to compare two different proprietary deuterium-modified compounds with the goal of…
Results from a new study indicate that mapping functions can be used with Cystic Fibrosis Questionnaire datasets to assess the utility values for interventions and costs related to Cystic Fibrosis. The study was recently published in the journal Health and Quality of Life Outcomes. Cystic fibrosis…
Researchers at the Research Centre for Medical Genetics in Russia and Abbott Laboratories in Russia and Germany have recently published in the Journal of Cystic Fibrosis their results on the efficacy and safety of pancreatic therapy in infants with exocrine pancreatic insufficiency (EPI) related…
Cystic fibrosis was ushered into the spotlight during President Barack Obama’s 2015 State of the Union Address as he highlighted the advances in CF therapy, which have served as a model for the President’s precision medicine initiative. Precision medicine paves the way for the development of therapies that are tailored…
A team of researchers at Aston University are currently working on understanding how bacteria present in children with a diagnosis of Cystic Fibrosis decreases their immunity, enabling other bacteria types to infect their airways. Cystic Fibrosis (CF) is a life-threatening inherited condition that affects approximately 9,000 people in the…
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