Vertex Pharmaceuticals said the worldwide COVID-19 outbreak has not affected its supply chain or its 2020 business outlook. The company said it’s confident it can continue to supply all its approved medications to patients with cystic fibrosis (CF) around the world. COVID-19 — an acronym for COrona…
cystic fibrosis
With the outbreak of coronavirus (COVID-19) affecting thousands of people around the world, individuals with respiratory conditions such as cystic fibrosis (CF) need to take precautions, according to medical expert Richard K. Mathis, MD. Mathis is a board member of Claire’s Place Foundation, a nonprofit organization that…
Eloxx Pharmaceuticals anticipates reporting topline data from the Phase 2 clinical trial program for ELX-02 — its lead therapeutic candidate to treat cystic fibrosis caused by nonsense mutations — during the first half of 2020, the company has announced. “We are committed to ensuring full enrollment of our Phase…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
In 1960, a 19-year-old woman with cystic fibrosis (CF) made medical history when she gave birth after only 34 weeks of pregnancy. The patient died three months after delivery. In 1986, by which time the life expectancy of people with the disease had risen substantially, some 50 women with…
Liver ultrasounds can help identify children with cystic fibrosis (CF) who are at higher risk of developing liver disease, a new study shows. The study, “Heterogeneous Liver on Research Ultrasound Identifies Children with Cystic Fibrosis at High Risk of Advanced Liver Disease: Interim Results of a…
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…
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