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,…
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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…
Synspira has started a Phase 1a clinical trial to determine the safety and tolerability of its cystic fibrosis therapy candidate SNSP113 in healthy volunteers and eventually in patients with stable CF. The dosing of the first participant marked the trial kick-off, the company announced. “SNSP113 is the first clinical [trial]…
Vertex Pharmaceuticals announced the start of a $500 million, 10-year charitable commitment to cystic fibrosis (CF) and other patients using its treatments, young adults studying science and mathematics, and communities across the U.S. The effort includes establishing a non-profit arm, The Vertex Foundation, with a $10 million initial investment to serve as a…
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
The first patient has been enrolled and dosed in the HOPE-1 Phase 2 clinical trial evaluating Spyryx Biosciences‘ investigational drug SPX-101 as a treatment for cystic fibrosis (CF). The HOPE-1 study (NCT03229252) will take place in Alberta and Ontario in Canada, the United Kingdom, France, and Portugal. Clinical…
Scientists have known for some time that cystic fibrosis patients have a variety of bacteria in their lungs. Researchers in Madrid have discovered two unusual predatory bacteria in a study investigating lung micro-organisms. Their study, “Individual Patterns of Complexity in Cystic Fibrosis Lung Microbiota, Including Predator Bacteria, over a 1-Year…
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