Clinical disappointment.

In tears tonight reading this. My hopes once ran sky high for this drug, that could potentially be the first to treat the underlying cause of Isaac's CF. 

For my beautiful boy and others with class 1 (stop/nonsense) mutations we must continue to campaign for research and treatments that target these rare (and reportedly more severe) mutations so that we are not left behind as the *untreatable few. 

*Only 10% of the 70,000 CF population worldwide have these kind of mutations.