Kalydeco update

[Rickengelage]

Hello guys,

Im Rick, 19 from the netherlands
I have cf, most common mutation, double DF508.
Infected with B.cepacia.

I recently saw that Vertex asked for Approval for Kalydeco in eu and Us for the most common mutation.

Great news! Hope it will be approved and be available soon!

Im wondering tho, has any of you guys had the opportunity to try Kalydeco already??

Who has been involved in studies?

I would really like to know about u guys and what it was like for u on this study? Or how its like now u can already try it? ( I heard U could get early access )

Hope to hear from anyone who had tried it!

Wish u all good health!

Rick.

 

[Dank]

I know they were doing trials on my mutation over 2 years ago, but still no luck for me. I have also been told by my doctor that only 2 mutations have the ability to be prescribed it, other than that it's a battle with insurance. That's coming from the Docs at Johns Hopkins here in Maryland. Can't help but wonder how cff.org is going to change this with the money it raised. I was also told that they are tryng to push for Kalydeco to be used for all class c/3 (I think??) genes, but they doubt it'll happen anytime soon.

 

[static]

Even the people in the clinical trials don't know if they are on the drug. If they are on it, they don't know at what dose. FDA needs accurate data, some subjects will get the short end of the stick. If you look hard enough I'm sure you can find both positive and negative stories about the drug like I did.

I'd just focus on keeping yourself as healthy as possible so when a cure does come you will have the lung function for it to make a difference.