Scientists at the University of California-Riverside have developed a novel way to help target therapeutics for cystic fibrosis (CF) and other diseases. Their study, “Inhibition of nonsense-mediated RNA decay by ER stress,” appeared in the journal RNA. The information encoded in human DNA is first converted to RNA, and…
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Once-a-day treatment with the antibiotic tobramycin does not harm the kidneys of patients with advanced cystic fibrosis (CF), a new study has found. Tobramycin taken three times a day was previously linked to acute and chronic kidney failure in CF patients. The study, “Glomerular and tubular renal function after repeated…
Rare mutations in the CFTR gene — which is defective in patients with cystic fibrosis (CF) — may occur in specific ethnic groups, suggests a new study that urges wider screening of mutations in patients who test negative for most previously identified mutations linked to CF. The study, “A rare CFTR mutation…
Certain bacteria may increase the risk of fungal colonization in cystic fibrosis (CF) patients, according to a study in Germany. CF makes a patient susceptible to chronic airway inflammation and infections, resulting in lung tissue damage. Scientists have long known that bacteria such as Pseudomonas aeruginosa and Staphylococcus aureus play roles…
Evaluating patients with cystic fibrosis (CF) before they board a flight may help predict whether they will need supplemental oxygen while in the air to prevent a severe decrease in blood oxygen levels, according to a study. The research, “Pre-Flight Evaluation Of Adult Patients With Cystic Fibrosis: A Cross-Sectional Study,” was…
Researchers are developing a compact respiratory device that will allow children whose lungs have failed from cystic fibrosis (CF) or other diseases to move about a hospital rather than having to stay in a bed during treatment or while awaiting a transplant. The Ambulatory Assist Lung for Children project…
Spyryx Biosciences successfully completed a Phase 1 clinical trial of its lead compound, SPX-101, in healthy volunteers and is now planning a Phase 2 study in patients with cystic fibrosis (CF). SPX-101 is an inhaled SPLUNC1-derived peptide created to regulate epithelial ion channels in the airways. The drug was…
Online vlogs — the video equivalents of web journals or blogs — can be an important platform for patients and families living with chronic diseases like cystic fibrosis (CF), a way of connecting with and learning from others in similar circumstances, researchers report. These vlogs, done by patients and addressing all aspects of life with a…
Three Stanford University School of Medicine researchers have won grants to advance the development of stem cell-based therapies, including gene editing, for cystic fibrosis (CF), autoimmune disorders and liver disease. The grants are part of $20.5 million awarded to 11 researchers by the California Institute for Regenerative Medicine (CIRM) during the current…
Galapagos dosed the first cystic fibrosis (CF) patient with GLPG2222, its novel CF corrector, in a Phase 2a clinical trial evaluating the drug as an add-on therapy to Kalydeco (ivacaftor) in CF patients harboring one F508del CFTR mutation and one gating mutation. The ALBATROSS Phase 2a clinical trial…
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