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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…

Guidelines for diagnosing cystic fibrosis (CF) were recently updated to give doctors firmer direction in determining signs of this disease, especially in those not diagnosed as newborns, and in being able to recommend the best treatment for each patient. The update, the work of 32 experts from nine countries, includes more information on mutations…

The top 10 priorities for cystic fibrosis research — set by CF patients and specialists alike — range from learning more about treatments that delay or prevent disease progression, to simplifying therapies so they’re less burdensome and getting patients to keep using a prescribed medication, according to a survey exercise led by scientists at The University…

A significant number of cystic fibrosis (CF) patients do not receive timely lung transplant evaluations because of their preference and their doctors’ possibly too-narrow focus, according to the results of a survey. The study explaining the results, “Cystic fibrosis physicians’ perspectives on the timing of referral for lung transplant…