Cystic Fibrosis News

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Inhaled Dry Powder Mannitol Improves Lung Function In CF

Adding inhaled dry powder mannitol to standard therapy for cystic fibrosis produced sustained improvement in lung function for up to 52 weeks, according to a new study. Along with the treatment's efficacy and good safety profile, the convenience and ease of administration of mannitol treatment may improve adherence with therapy in these patients.

In the double-blind study, which was supported by Pharmaxis Limited, 318 patients were randomized to treatment with 400 mg bid inhaled mannitol or 50 mg bid inhaled mannitol (control group) for 26 weeks, followed by an additional 26 weeks of open-label active treatment.

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New Drug Candidates Discovered For Cystic Fibrosis And Other Diseases

A new discovery by Californian scientists may lead to a pharmaceutical breakthrough for a wide range of illnesses that involve the hydration of cells that line the inner surfaces of our body's organs and tissues. In a new report appearing in the FASEB Journal*, the researchers describe how they used high-throughput screening to identify small-molecule drug candidates which help cells bypass defective channels that normally move salt and water through cell membranes. These drug candidates work by activating an alternative chloride channel called "TMEM16A" that might be effective in treating conditions such as cystic fibrosis, dry eye and dry mouth diseases and slow-transit constipation.

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An 'Unconventional' Path To Correcting Cystic Fibrosis

Researchers have identified an unconventional path that may correct the defect underlying cystic fibrosis, according to a report in the September 2nd issue of the journal Cell, a Cell Press publication. This new treatment dramatically extends the lives of mice carrying the disease-associated mutation.

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