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View Full Version : Does anyone know if Kalydeco would work or can be tried on other mutation?



BleedOrange1968
03-10-2012, 10:40 PM

BleedOrange1968
03-10-2012, 10:40 PM

Anomie
03-10-2012, 10:56 PM
It works slightly with other mutations but its better when taken in conjunction with 661 or 809.

Anomie
03-10-2012, 10:56 PM
It works slightly with other mutations but its better when taken in conjunction with 661 or 809.

saveferris2009
03-11-2012, 12:44 AM
Orange, there's a lot left to be discovered about Kalydeco as a mono therapy. So far, it's only been tested in DDf508 and G551D. That's why many people are trying the drug, even if they don't have G551D. It will be exciting to see what people report after trying the med. And some insurances are paying for it. The amount of CFTR that a gene can create can be influenced by a lot of things - modifier genes, exercise, the food we eat. So Kalydeco may have different impacts on different people....

saveferris2009
03-11-2012, 12:44 AM
Orange, there's a lot left to be discovered about Kalydeco as a mono therapy. So far, it's only been tested in DDf508 and G551D. That's why many people are trying the drug, even if they don't have G551D. It will be exciting to see what people report after trying the med. And some insurances are paying for it. The amount of CFTR that a gene can create can be influenced by a lot of things - modifier genes, exercise, the food we eat. So Kalydeco may have different impacts on different people....

Keepercjr
03-11-2012, 08:33 PM
I left a message for the CF nurse that I want to try it and that I'm not going to accept a "no". If they try to tell me no I have a clinic appt on Wednesday and I will make myself very clear.

Keepercjr
03-11-2012, 08:33 PM
I left a message for the CF nurse that I want to try it and that I'm not going to accept a "no". If they try to tell me no I have a clinic appt on Wednesday and I will make myself very clear.

saveferris2009
03-11-2012, 08:54 PM
You go girl!
Nothing is guarenteed, of course. It's a long shot. But it's certainly worth a try.....

saveferris2009
03-11-2012, 08:54 PM
You go girl!
Nothing is guarenteed, of course. It's a long shot. But it's certainly worth a try.....

Kristen
03-11-2012, 10:11 PM
In vitro studies have shown that it works on all gating mutations. It may also work on other mutations (specifically Class IV & V mutations) but studies have not been published.

Kristen
03-11-2012, 10:11 PM
In vitro studies have shown that it works on all gating mutations. It may also work on other mutations (specifically Class IV & V mutations) but studies have not been published.

BleedOrange1968
03-11-2012, 11:11 PM
I am f508 and 3272-26a>g. How do I know if mine is gating and what class I'm in??
Darryl 44 dx 24

BleedOrange1968
03-11-2012, 11:11 PM
I am f508 and 3272-26a>g. How do I know if mine is gating and what class I'm in??
Darryl 44 dx 24

saveferris2009
03-12-2012, 01:44 AM
http://lmgtfy.com/?q=3272-26a%3Eg

looks like a splicing mutation

saveferris2009
03-12-2012, 01:44 AM
http://lmgtfy.com/?q=3272-26a%3Eg

looks like a splicing mutation

Anomie
03-12-2012, 02:06 AM
Ask your doctor to write you a prescription for it and test it out. Your mutation is probably too rare for them to run a clinical trial on and if your doctor writes you a prescription then your insurance company will probably pay for it. Some other people on this site have been doing that exact same thing with some pretty positive results!

Anomie
03-12-2012, 02:06 AM
Ask your doctor to write you a prescription for it and test it out. Your mutation is probably too rare for them to run a clinical trial on and if your doctor writes you a prescription then your insurance company will probably pay for it. Some other people on this site have been doing that exact same thing with some pretty positive results!

jessykt
03-12-2012, 03:34 PM
I am on Kalydeco and have DeltaF508 and G551D. I was told that Kalydeco works MILDLY with other mutations, but the most success was with G551D. The reason why it's being used only for G551D is because to pass FDA regulations, it had to benefit a certain percentage of population. Now that it's been passed and may be prescribed for G551D, they can start working their way backward with other mutations.

jessykt
03-12-2012, 03:34 PM
I am on Kalydeco and have DeltaF508 and G551D. I was told that Kalydeco works MILDLY with other mutations, but the most success was with G551D. The reason why it's being used only for G551D is because to pass FDA regulations, it had to benefit a certain percentage of population. Now that it's been passed and may be prescribed for G551D, they can start working their way backward with other mutations.

jessykt
03-12-2012, 03:36 PM
While I understand your perserverance, I highly doubt you'll be able to receive the drug. Since you do not have the G551D, Vertex wouldn't even consider sending the drug, let alone your insurance covering the $294,000 year price tag. To sign up for Kalydeco, my doctors had to send in my verification that I did indeed have G551D.

jessykt
03-12-2012, 03:36 PM
While I understand your perserverance, I highly doubt you'll be able to receive the drug. Since you do not have the G551D, Vertex wouldn't even consider sending the drug, let alone your insurance covering the $294,000 year price tag. To sign up for Kalydeco, my doctors had to send in my verification that I did indeed have G551D.

Hardak
03-12-2012, 05:43 PM
currently trials of this on DF508X are being set up, they are using this and something else (sorry i forget what exactly) in cunjunction. the DF508X mutations protines simply don't make it to the cell wall, its been known for years how to moblize the protines but not activate them at the cell wall, which Kalydeco is suppose to do. (if this is what I think it is).

Hope this info helps.

Hardak
03-12-2012, 05:43 PM
currently trials of this on DF508X are being set up, they are using this and something else (sorry i forget what exactly) in cunjunction. the DF508X mutations protines simply don't make it to the cell wall, its been known for years how to moblize the protines but not activate them at the cell wall, which Kalydeco is suppose to do. (if this is what I think it is).

Hope this info helps.

Keepercjr
03-12-2012, 11:57 PM
I don't know if I'll be able to see this since I can't seem to see the end of the thread???
Anyway, my CF nurse called back this evening. She said that the vertex rep told her today that the only way insurance will cover it is if you have the right mutation otherwise you have to pay out of pocket. I said that I know for a fact that others with different mutations have gotten it no problem through their insurance. So I asked to please prescribe it and see if the insurance will cover it. She said sure and that she will leave a note for the other CF nurse who will be in tomorrow. I can see now that that even the reps for the DRUG COMPANY (!) don't know what is going on. I'm crossing my fingers!!! I have Medco btw for prescription coverage.

Keepercjr
03-12-2012, 11:57 PM
I don't know if I'll be able to see this since I can't seem to see the end of the thread???
Anyway, my CF nurse called back this evening. She said that the vertex rep told her today that the only way insurance will cover it is if you have the right mutation otherwise you have to pay out of pocket. I said that I know for a fact that others with different mutations have gotten it no problem through their insurance. So I asked to please prescribe it and see if the insurance will cover it. She said sure and that she will leave a note for the other CF nurse who will be in tomorrow. I can see now that that even the reps for the DRUG COMPANY (!) don't know what is going on. I'm crossing my fingers!!! I have Medco btw for prescription coverage.