kalydeco question.

[Angel2393]

This may seem like a stupid question, but I hardly ever speak to my CF team anymore as I have had a transplant 5 years ago, so we just focus on my rejection medications and etc...but, I have the one gene needed for the kalydeco, must you have 2 to take it?

 

[Aboveallislove]

NO!!! If you have one copy of 551 it is "on label". If you have one copy of a gating mutation it will likely work and you likely will be able to get off label. I think only 1 or 2 folks in the Phase 3 trial had 2 copies of 551--so the benefit was so great even though only 1 copy.