Wednesday, February 23, 2011

So it goes

Today the NYT published an article about phase 3 clinical trial results for VX-770, a Vertex drug in development for treatment of the underlying genetic defect that causes cystic fibrosis.  The results are very promising -- both in the specific improvements in lung function and other measures, but, perhaps more importantly, in demonstrating that the drug is acting on the sodium transport defect that is at the root of CF.  One of the main ways CF is diagnosed is through sweat tests.  Will used to repeat the story of an old Swiss/German folk saying, something like: "Sad the child who tastes salty when kissed, for he is cursed and soon must die." People taking the the Vertex drug had less salt in their sweat.  Much less. 

This particular medication is desinged to help one of the less common CF gene mutations; but Vertex has a similar medication in development, based in part on this research, that would treat the most common mutation; the one Will had.  It's a hopeful day for the CF community.  Hopeful.

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