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…
Researchers have identified a signal that controls when lung bacteria in cystic fibrosis patients start producing biofilm — a sticky secretion that protects them from attack and makes the bacteria virtually impossible to get rid of. Since the signal suppresses bacteria’s biofilm-making machinery, the finding could lead to treatments that…
A cystic fibrosis expert will be among more than 80 speakers at this month’s Rare Disease & Orphan Products Breakthrough Summit in Washington. The nonprofit National Organization for Rare Disorders (NORD) will be hosting the sixth annual event Oct. 16-17 at the Marriott Wardman Park Hotel. It is expected to attract…
Researchers have discovered that the Bpifb1 gene plays an important role in controlling levels of a mucin protein called MUC5B, one of the main components of mucus. This means that the gene also plays a crucial role in protecting the body’s airways. The team found that deleting the Bpifb1 gene in…
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