Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported.
News
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar. William Whitman…
The National Institute for Health and Care Excellence (NICE) in the U.K. is currently in the process of evaluating Vertex Pharmaceuticals’ triple combination therapy — known as Trikafta in the United States — for the treatment of cystic fibrosis (CF). The institute expects to deliver its…
Two years ago, Israel became only the fourth country in the world — after the U.S., Germany, and Belgium — to agree to underwrite the cost of Vertex Pharmaceuticals’ three available therapies for cystic fibrosis: Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combo). But that’s not…
To put it in yoga-speak, instant karma greeted the co-founders of CF Yogi — Myra Giaffoglione of Glenwood, Iowa, and Elaine Malik of Seattle — when they spoke for the first time in the spring of 2018. Malik, 40, and Giaffoglione’s two kids, 7-year-old Jack…
The U.S. Food and Drug Administration (FDA) has granted its orphan drug designation to ARV-1801 (sodium fusidate tablets), a treatment developed by Arrevus for patients with cystic fibrosis (CF). CF patients frequently develop lung infections due to the accumulation of thick mucus in their lungs, creating…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
More than 70% of home nebulizers used by children with cystic fibrosis (CF) are contaminated with microorganisms, posing a serious potential health risk to patients and underscoring a significant need for educational programs focused on nebulizer maintenance and hygiene, a research study found. The study also found a…
The Cystic Fibrosis Foundation has reached an agreement with Synspira Therapeutics to support the further development of SNSP003, an enzyme replacement therapy (ERT) designed to treat malabsorption syndromes, including those affecting patients with cystic fibrosis (CF). Malabsorption syndromes are a group of disorders that develop when there…
A new non-invasive tool, called XV technology, allows professionals to visualize airflow in a living lungs and could help in the diagnosis, monitoring, and treatment of cystic fibrosis (CF) and other respiratory lung diseases, scientists report. The technology was developed by researchers at Monash University, and has been patented by the…
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