News

Vertex Pharmaceuticals Inc. recently presented preliminary study results of FDA-approved Kalydeco for the treatment of cystic fibrosis (CF) in combination with an investigational drug. Despite the fact that the combined therapy helped improve breathing in CF patients, the outcomes were less significant than expected by analysts, which disappointed investors. Vertex conducted…

When Julie Goodrow‘s first child was born in 2004, she never imagined that her life would be so different from other new mothers. Just one day after being born, the doctors told Julie that her daughter, Elena, now 11 years old, might suffer from a chronic and progressive disease, but it was…

Photo of the the Narberth Cystic Fibrosis Run The Narberth Cystic Fibrosis Run will celebrate its 20th year on April 25th, with proceeds to fund granting wishes to children suffering from CF and their families. To date, the Take A Breather…

Anthera Pharmaceuticals recently announced that it received a $3 million award from the Cystic Fibrosis Foundation Therapeutics to support both the manufacturing and clinical development of Sollpura™ (liprotamase), Anthera’s new pancreatic enzyme replacement therapy. Cystic Fibrosis Foundation Therapeutics is an affiliate of the Cystic Fibrosis Foundation, a non-profit organization. Anthera Pharmaceuticals is a…

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,…

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…