Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
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About 4,400 researchers, doctors, patients and others from 46 countries are converging on Indianapolis for the 31st Annual North American Cystic Fibrosis Conference — the world’s largest gathering dedicated to cystic fibrosis research and care. The Nov. 2-4 event at the Indiana Convention Center will feature more than 350 sessions and…
Polyphor and the European Innovative Medicines Initiative (IMI) will fund up to 10 million euros (around $11.6 million U.S.) to support and advance the development of an inhaled formulation of Polyphor’s investigational antibiotic murepavadin (POL7080). Polyphor is investing 5 million euros into the development project; the IMI will provide up…
A new tool allows researchers and doctors to visualize a 3D model of a cystic fibrosis (CF) lung that incorporates information about microbes, which may help improve targeted drug delivery, a new study shows. The study, “Three-Dimensional Microbiome and Metabolome Cartography of a Diseased Human Lung,”…
The 2018 spring scholarship application deadline for the United States Adult Cystic Fibrosis Association (USACFA)‘s Lauren Melissa Kelly Scholarship program has been extended to Nov. 15, 2017. The scholarships, awarded each spring and fall semester, are open to adults with cystic fibrosis (CF) who are…
People with cystic fibrosis (CF) may be able to avoid having to travel to speciality clinics if health professionals can monitor them via phone or video messaging, says the National Institute for Health and Care Excellence (NICE) in is first Cystic Fibrosis Guideline. In this new guideline,…
AzurRx BioPharma is planning to expand the clinical development of its investigative therapy MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) caused by chronic inflammation of the pancreas and cystic fibrosis (CF) into Europe. The company has submitted an Investigational Medicinal Product Dossier (IMPD) — similar to an…
Cystic Fibrosis Ireland has started a “Fight to Breathe” campaign featuring a 60-second film that will be shown at 73 movie theaters across the country for five weeks. The awareness effort will let people know that living with cystic fibrosis literally means having to fight for every breath. The public service…
The Institute for Clinical and Economic Review (ICER) is planning to evaluate and compare the clinical effectiveness and value of cystic fibrosis treatments now available or awaiting approval, with a tentative focus on several by Vertex Pharmaceuticals. According to information provided in an ICER press release, the institute intends…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
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