cystic fibrosis

The American Thoracic Society (ATS) recommends the use of home oxygen therapy in children with cystic fibrosis (CF) who have mild or severely reduced oxygen saturation, according to the most recent guidelines. ATS clinical guidelines for home oxygen therapy use in pediatric patients with lung and pulmonary vascular…

Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the correct functioning of the CFTR protein, the therapy’s developer announced. With a pill taken twice a day, Kalydeco, by Vertex Pharmaceuticals, makes it possible to resolve the underlying…

The Horizon 2020 research and innovation program of the European Union granted €6.8 million (approximately $7.7 million) to an international research team for the development of non-viral gene delivery technology. This new gene therapy strategy has the potential to improve the health of patients with genetic diseases worldwide, including…

The European Commission has approved the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals, the therapy’s manufacturer, announced. Orkambi was previously approved in Europe to treat CF patients 6 and older with the…