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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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…
A Cystic Fibrosis Foundation senior vice president is a finalist for the Woman of the Year Award that the Washington Women in Public Relations is scheduled to present today, Friday, Nov. 17. The finalists besides the foundation’s Amy DeMaria are Kathy Baird, Ogilvy & Mather’s managing director of content and social media for North America,…
All mice with cystic fibrosis that received iclaprim for a pneumonia infection survived, according to a study. Motif Bio reported the finding, which involved the Staphylococcus aureus bacteria, and the results of another preclinical-trial study at the IDWeek 2017 conference in San Diego, Oct. 4-8. Iclaprim is designed to kill bacteria that develop resistance…
Experiments in live pigs show that the genetic mutation that causes cystic fibrosis (CF) prevents secretion of airway surface liquid when bacteria enter the lungs — a mechanism that may, at least in part, explain why CF patients are not able to ward off bacterial infections. Researchers at the…
A research team at Imperial College London (ICL) has developed a quick, cheap method to detect the presence of Pseudomonas aeruginosa in sputum samples collected from patients with cystic fibrosis (CF). Using small engineered DNA circuits called cell-free biosensors, the method reveals molecules specifically produced by P. aeruginosa. When…
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