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…
News
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…
The Dayton Children’s Hospital, an institution dedicated to advancing treatment for cystic fibrosis (CF), has been granted funding from the Cystic Fibrosis Foundation for the hospital to expand its studies and find an effective treatment for the disease. The grant will be invested in hiring more research coordinators and…
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…
N30 Pharmaceuticals, Inc., a clinical-stage pharmaceutical firm committed to developing product candidates to address cystic fibrosis (CF), recently announced that the company has changed its name to Nivalis Therapeutics, Inc. Nivalis Therapeutics’ lead product candidate is N91115, a new inhibitor of S-nitrosoglutathione reductase (GSNOR) for the treatment of CF. The company’s new name, Nivalis…
A study led by researchers at West Virginia University presented a case report of a child who was diagnosed with cystic fibrosis (CF) based on an intussusception manifestation. The study was recently published in the Journal of Pediatric Surgery Case Reports and is entitled…
An article published in the Journal of Clinical Investigation provides evidence that challenges the previously held assumptions as to where the abnormal accumulation of sticky mucus, a key feature in cystic fibrosis (CF), originates. The study entitled “Defective goblet cell exocytosis contributes to murine cystic…
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