Kalydeco and D1152h

[Sophiesmum]

Hi all!

just wondered if anyone has managed to get kalydeco off label with this mutation? And if so what are your experiences?

many thanks!

Gemma x

 

[GenH]

Hi all!

just wondered if anyone has managed to get kalydeco off label with this mutation? And if so what are your experiences?

many thanks!

Gemma x

Hi Gemma, I do not know anyone off label with this mutation, but if you haven't already seen these graphs, they show that Kalydeco should help D1152H significantly. The graphs would be strong evidence in terms of accessing Kalydeco off label (if you are in the US): http://sixtyfiverosesblog.wordpress.com/kalydeco-with-other-mutations/

 

[Sophiesmum]

Hiya!

thanks for your reply Yep I've been stalking your blog we are in the UK so can only get once it's been approved-big boo! I know there's a residual mutation trial ongoing currently and waiting on results I guess to see what happens! Sophies other mutation is DF508 so I'm wondering whether the combo will come before trying just purely kaly alone in her case if that makes sense, as it's further along the trial route. I was curious really as to whether anyone with her second mutation had managed to try out kaly on its own.

thanks again! X

 

[jamieharris]

Useful thread. Thanks for sharing