cystic fibrosis

Ultrasound imaging can be a reliable diagnostic tool for early detection of liver disease in children with cystic fibrosis (CF), a study finds. Using this strategy can help clinicians identify CF patients most at risk for liver damage caused by cirrhosis (tissue scarring), researchers suggest. These findings were reported in…

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…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

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…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

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…

EffRx Pharmaceuticals announced that it has signed an exclusive license agreement with Pharmaxis to register and commercialize Bronchitol (mannitol) for the treatment of cystic fibrosis (CF) in Switzerland. According to EffRx, Bronchitol should be available to Swiss patients by 2021. Bronchitol is a dry powder for…

Murepavadin (POL7080), Polyphor‘s lead investigational antibiotic targeting Gram-negative bacteria, behaved as expected and was well-tolerated when administered to mice harboring a lung infection caused by Pseudomonas aeruginosa, data from a preclinical study show. Of note, P. aeruginosa is a bacteria that often infects the lungs of patients…