A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
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The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of children, ages 6 to 11 years, with cystic fibrosis (CF) who have certain mutations that respond to treatment, its developer, Vertex Pharmaceuticals, has announced. In order…
Long-term use of inhaled antibiotics, given to treat lung infections caused by the bacteria Pseudomonas aeruginosa in people with cystic fibrosis (CF), can for young children raise the risk of a new infection by Aspergillus fungi, a study suggests. The study, “Pseudomonas…
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Treatment with Orkambi significantly improves exercise tolerance in adult cystic fibrosis patients with severe disease as early as 4 weeks after treatment — as shown by results of the six-minute walk test (6MWT), a study found. The findings support the use of exercise tolerance as an important…
Small proteins with antimicrobial properties found in the mucus of the brown garden snail may open the door to the development of new treatments for patients with cystic fibrosis (CF) who often experience lung infections caused by the bacteria Pseudomonas aeruginosa, a study says. The findings of the…
If the Cystic Fibrosis Trust (CFT) has its way, the United Kingdom will be awash in yellow on June 21. On that day, the nonprofit organization is asking patients and supporters to don yellow — hopefully while fundraising — to symbolize a brighter future for those with the progressive inherited…
Kalydeco (ivacaftor) preserved lung function, improved the nutritional status, and reduced hospital visits and chronic bacterial infections in people with cystic fibrosis (CF) when administered for up to five years in a clinical setting, a long-term, real-world, observational study showed. The study, “Disease progression in patients…
The experimental treatment eluforsen is safe for adults with cystic fibrosis (CF) caused by a mutation called F508del, a new study reports, supporting further investigation and development of the treatment. The study, titled “Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic…
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