Article title: Patient personalized translational tools in cystic fibrosis to transform data from bench to bed-side and back Authors: Kavisha Arora, Fanmuyi Yang, John Brewington, Gary McPhail, Alexander R. Cortez, Nambirajan Sundaram, Yashaswini Ramananda, Herbert Ogden, Michael Helmrath, John P. Clancy,…
Scientists created a potentially powerful new strategy for treating cystic fibrosis and potentially other diseases; it involves small, nucleic acid molecules called oligonucleotides that can correct some of the gene defects that underlie CF but are not addressed by existing modulator therapies.
In their lab, CRCHUM scientists Emmanuelle Brochiero and Damien Adam are studying ways of better predicting the efficacy of medications and helping injured lungs repair and regenerate.
A Northwestern University-led research team has developed a novel skin-mounted sticker that absorbs sweat and then changes color to provide an accurate, easy-to-read diagnosis of cystic fibrosis within minutes.
An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90 percent of the cystic fibrosis population in the United States.
Staphylococcus aureus (which includes MRSA) is the most prevalent organism isolated from the airways of children with cystic fibrosis (CF), and is treated using antibiotics, but its role in lung disease is poorly understood
DALLAS – Nov. 4, 2020 – A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients’ lives.
An up-and-coming gene therapy for blood disorders. A new class of medications for cystic fibrosis. Increased access to telemedicine. These are some of the innovations that will enhance healing and change healthcare in the coming year, according to a distinguished panel of clinicians and researchers from Cleveland Clinic.
In conjunction with the 2020 Medical Innovation Summit, Cleveland Clinic announced the Top 10 Medical Innovations for 2021.
This research shows that both Pseudomonas and Burkholderia use toxic weaponry, called Type VI Secretion Systems (T6SS), to compete with and establish dominance over each other. It could be possible to target or mimic this weaponry to defeat the bacteria before they cause irreparable lung damage.
• Studies conducted in mouse models, patients with cystic fibrosis, and different types of cells have helped to define how cystic fibrosis affects the kidneys.
• A urine test may help to assess aspects of cystic fibrosis in patients, which may be useful for testing the effectiveness of new medications.
The Academy of Nutrition and Dietetics has released a comprehensive guideline to help registered dietitian nutritionists address the needs of people with cystic fibrosis who are at risk of developing nutrition problems commonly associated with pulmonary disorders.
Article title: Resveratrol restores intracellular transport in cystic fibrosis epithelial cells Authors: Binyu Lu, Deborah A. Corey, Thomas J. Kelley From the authors: “Though further studies are necessary, the ability of resveratrol to reverse [cystic fibrosis] cellular phenotypes in a manner not…
Nationally-known developer Chris Jeffries and his wife Lisa have donated $25 million to Henry Ford Health System, the largest single gift from an individual in the health system’s 105-year history. This historic gift will rapidly accelerate the growth and expansion of Henry Ford’s Precision Medicine program, with the ultimate goal of creating a Precision Health Center. The efforts will have a robust focus on the advancement of cancer research and treatment, while also expanding to other medical specialties treating behavioral health, cardiovascular and metabolic diseases.
Article title: Inhibition of ATP hydrolysis restores airway surface liquid production in cystic fibrosis airway epithelia Authors: Catharina van Heusden, Brian Button, Wayne H. Anderson, Agathe Ceppe, Lisa C. Morton, Wanda K. O’Neal, Hong Dang, Neil E. Alexis, Scott H.…
People with cystic fibrosis who carry genetic variants that lower RNF5 gene expression have more mutant CFTR protein on cell surfaces. Even if the CFTR protein isn’t fully functional, it’s better than none, and may explain why some with cystic fibrosis are less prone to infection than others.
A phase three clinical trial that UT Southwestern participated in determined that a three-drug combination improved lung function and reduced symptoms in cystic fibrosis (CF) patients who have a single copy of the most common genetic mutation for the disease.
Steve Rowe, M.D., professor of medicine at the University of Alabama at Birmingham and director of the Gregory Fleming James Cystic Fibrosis Research Center, is available to discuss the FDA approval of Trikafta, a new triple combination therapy for CF. …