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November study found Kalydeco improved lung function, reduced complications in patients with cystic fibrosis who have G551D mutation

The new drug Kalydeco is being hailed as a breakthrough in the treatment of cystic fibrosis, targeting the underlying genetic cause of the disease decreasing the symptoms. Dr. Jon LaPook reports.

People with an uncommon form of cystic fibrosis started gaining weight and were better able to breathe than their untreated counterparts after just two weeks on an experimental drug, according to a study published November 2 in The New England Journal of Medicine . If approved by the U.S.

In 1989 when scientists discovered the defective gene that causes cystic fibrosis, a serious hereditary disorder that primarily strikes children of European descent, it seemed as though a long-hoped-for cure might soon follow. After all, tests in many laboratories showed that providing normal copies of the gene should enable patients to make healthy copies of the protein specified by the gene. If successful, that feat would go a long way toward restoring health in the tens of thousands of people around the world who suffered from cystic fibrosis and typically died in their late 20s

By Heidi Ledford of Nature magazine Early next year, a drug for cystic fibrosis is expected to come before the US Food and Drug Administration for approval. [More]

A series of new video games for kids with cystic fibrosis not only helps them stick to treatment regimens, but also significantly improves breathing performance. Gamification to the rescue! One of the hardest parts of treating cystic fibrosis in children is convincing them to perform tedious breathing exercises required to keep airways clear. However, a new study that applies gamification techniques to cystic-fibrosis treatment indicates that specially made video games not only get children to perform breathing exercises--they also improves breathing performance when not playing games