One F508 + one rare mutation

[mmoders]

Will any of the new meds in the pipeline help with just one F508 mutation? The other one is a rare mutation. Or will it only help those with DD508?

 

[aannddd]

If you read the vertex releases in the phase 2 studies for 809, it helped for those with one copy of deltaF508, but not as significant as those with 2 copies of deltaf508. I am guessing they are doing continued trials for those with the 2 copies to help ensure that hey can get the drug through the initial FDA approval. Once it is approved, I think there will be follow-up studies for those with only one copy. I feel you frustration, my son only has one copy of DeltaF508.