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Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for cystic fibrosis (CF), the therapy’s developer, Translate Bio, has announced. Fast track is given to medications that aim to treat serious or life-threatening conditions and…

Enrollment is complete for a European initiative to test an organoid-based, personalized-medicine approach to treating cystic fibrosis, with the first trial starting later this year, said Proteostasis Therapeutics (PTI) and CF Europe. The Human Individualized Therapy of CF (HIT-CF) initiative will test PTI drug combinations through…

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.

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…