A new study revealed that microRNA16 — a small non-coding RNA genes that regulates gene expression — can restore the F508del-CFTR protein function in airway cell lines and primary cultures of differentiated human bronchial epithelia from F508del homozygotes that express mutant CFTR protein endogenously. The finding is significant, as…
Researchers at the University of Arkansas for Medical Sciences and the Department of Health, Arkansas reported a rare case of pulmonary tuberculosis in a cystic fibrosis (CF) patient. The report, which was published in the North American Journal of Medical Sciences and is entitled “…
The U.S. Food and Drug Administration on Thursday, July 2 announced that it has approved the first cystic fibrosis treatment that targets the cause of the disease. Orkambi is an oral ivacaftor/lumacaftor combination medication for treatment of CF that targets the underlying cause of the disease in people who have…
Researchers at Stanford University School of Medicine recently reported that Hispanic patients with cystic fibrosis (CF) have a higher mortality rate than non-Hispanic patients. The study was published in the journal Chest and is entitled “Assessing differences in mortality rates and risk factors…
Accuracy and efficiency are of utmost importance when deciding on a diagnostic tool to detect cystic fibrosis in newborns. The gold standard of detection is measuring sweat ion concentration, as patients with defect CFTR proteins have an elevated electrolyte content in their sweat. However, another method that is now only…
The National Association for Stock Car Auto Racing, otherwise known as NASCAR, is hosting a race this Saturday to celebrate July 4th at the Chicago Speedway. Millions of fans will be tuned in, and the Cystic Fibrosis Foundation is set to make a nationwide statement through ARRIS-backed NASCAR driver Daniel Suarez, whose…
ManRos Therapeutics SA and Cyclacel Ltd, an exclusively owned subsidiary of Cyclacel Pharmaceuticals, Inc., recently announced a partnership, supply and licensing agreement for the commercialization and development of Cyclacel’s oral seliciclib capsules by ManRos for the treatment of patients with cystic fibrosis (CF). Under the terms…
ProQR Therapeutics N.V. a company developing RNA medicines for the treatment of severe diseases including cystic fibrosis, recently announced that it has initiated the enrollment of patients into PQ-010-001, a Phase 1b clinical trial for QR-010. The compound is a new RNA-based therapeutic drug designed to restore the genetic mutation in the mRNA…
Researchers at the Hellenic Cancer Society and the Biomedical Research Foundation, Academy of Athens in Greece conducted a review on exhaled breath condensate analysis as a tool to assess lung function in patients with respiratory disorders. The study was recently published in The Scientific World…
Jason van’t Slot — a 20-year-old South African — was born with Cystic Fibrosis, and after receiving a prognosis of a 10-year life span at the age of 8 months, celebrated his 20th year of living with CF by cycling the beguiling and grueling ABSA Cape Epic — the toughest…
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