a) i'm female... b) i dont know why my nurse described it that way.. she just did... and c) yes I am homozygous delta f508. I have been geneticaly tested and i'm positive that my doctors would not lie to me about what mutations i havce. plus if i was homozygous... and provided proof... i wouldn't have been able to participate in the study....so yeah
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DF508ers who participated in VX-770 trials
- Thread starter jmiller
- Start date
a) i'm female... b) i dont know why my nurse described it that way.. she just did... and c) yes I am homozygous delta f508. I have been geneticaly tested and i'm positive that my doctors would not lie to me about what mutations i havce. plus if i was homozygous... and provided proof... i wouldn't have been able to participate in the study....so yeah
saveferris2009
New member
I will get a hold of this drug and let you know how I feel.
I am hetero, with 2 class II's.
I am hetero, with 2 class II's.
saveferris2009
New member
I will get a hold of this drug and let you know how I feel.
I am hetero, with 2 class II's.
I am hetero, with 2 class II's.
C
cfsucks
Guest
^ what do you mean? do you carry df508? how will you get a hold of it? do you carry a gating mutation?
C
cfsucks
Guest
^ what do you mean? do you carry df508? how will you get a hold of it? do you carry a gating mutation?
No he doesn't carry a gating mutation because he says he's got two class 2 mutations which involves degradation of the protein before reaching the cell surface. I think he means he's heterozygous for DF508 in which case he might be trying to get on the vx 809 trial or maybe he's just blowing smoke up our yoo-hoos.
No he doesn't carry a gating mutation because he says he's got two class 2 mutations which involves degradation of the protein before reaching the cell surface. I think he means he's heterozygous for DF508 in which case he might be trying to get on the vx 809 trial or maybe he's just blowing smoke up our yoo-hoos.
Incomudrox
New member
Amy doesn't blow smoke. <img src="i/expressions/face-icon-small-wink.gif" border="0">
Incomudrox
New member
Amy doesn't blow smoke. <img src="i/expressions/face-icon-small-wink.gif" border="0">
saveferris2009
New member
I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely.
So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me.
You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available).
So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?"
I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot.
As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works.
And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.
So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me.
You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available).
So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?"
I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot.
As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works.
And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.
saveferris2009
New member
I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely.
So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me.
You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available).
So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?"
I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot.
As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works.
And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.
So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me.
You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available).
So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?"
I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot.
As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works.
And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.
C
cfsucks
Guest
<div class="FTQUOTE"><begin quote><em>Originally posted by: <strong>saveferris2009</strong></em> I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely. So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me. You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available). So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?" I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot. As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works. And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.</end quote>
this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a while but i have good insurance coverage so i will see if i can possibly try to get it as well.
keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?
this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a while but i have good insurance coverage so i will see if i can possibly try to get it as well.
keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?
C
cfsucks
Guest
<div class="FTQUOTE"><begin quote><em>Originally posted by: <strong>saveferris2009</strong></em> I have insurance that doesn't require approval for any drug. Any drug that a doc prescribes, I can get. Yes, I am extremely blessed - this is one of the reasons I chose the job that I have. I thank God for this blessing, absolutely. So I'm going to try Kalydeco for myself, even though I don't have a gating mutation. Even a tiny improvement, however small, is worth it to me. You have to remember that when doing a clinical trial, the goal for a drug company is to maximize the chances of FDA approval. I don't work for Vertex nor do I speak for them. But it's important to understand that just because Vertex didn't pursue FDA approval for DF508 doesn't mean it didn't work. D551G showed incredible results, therefore this presented the best chance of getting FDA approval. Once a drug is on the market, docs can Rx for any reason they please (doesn't mean insurance will pay for it, of course, but the point is that the drug is available). So it's obvious that Kalydeco doesn't have the same impact on DF508 that it has on G551D or any gating mutation for that matter. BUT that doesn't mean it won't have any impact. Some of the phase II trials show otherwise. To me this doesn't represent false hope - it represents trying something out. If I see some improvement, great. If not, at least I tried. I'll never wonder "hmmmmm..... what impact would this drug have had on me?" I try stuff. Like I tried NAC to see if it would work. Like I tried allergy shots. Like I tried cromolyn sodium. Like I tried denufosol. Like I tried Magnesium supplements. Some of it fails, some if it works. But much of what I try has little downside, so for me, it's worth a shot. As Dr. Warwick told me when I visited him in 2006 - every CFer is a scientist, doing experiments to understand how this disease works. And yes, I don't blow smoke. Well, I blow pulmozyme smoke. But that's about it.</end quote>
this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a while but i have good insurance coverage so i will see if i can possibly try to get it as well.
keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?
this is very interesting- i'm in canada which is unfortunate so we won't see kalydeco for a while but i have good insurance coverage so i will see if i can possibly try to get it as well.
keep us updated on your own trial- are you going to measure your improvement with clinical results, or just how you feel? will you be attempting to get sweat chloride tests and so forth done?
Amy, I'm impressed that your doc is willing to prescribe it for you. My insurance says they will cover it off-label, but my doc was still hesitant to prescribe it for me, even though I have R117H.
(He said he didn't want to be the roadblock to me getting it, but he was concerned about it not being approved for my mutation (yet) and it being so expensive. He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)
(He said he didn't want to be the roadblock to me getting it, but he was concerned about it not being approved for my mutation (yet) and it being so expensive. He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)
Amy, I'm impressed that your doc is willing to prescribe it for you. My insurance says they will cover it off-label, but my doc was still hesitant to prescribe it for me, even though I have R117H.
(He said he didn't want to be the roadblock to me getting it, but he was concerned about it not being approved for my mutation (yet) and it being so expensive. He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)
(He said he didn't want to be the roadblock to me getting it, but he was concerned about it not being approved for my mutation (yet) and it being so expensive. He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)
saveferris2009
New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Kristen</b></i> but he was concerned about it not being approved for my mutation (yet) </end quote>
Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost....
<div class="FTQUOTE"><begin quote>and it being so expensive.</end quote>
He's not paying for it. Why does he care?
I hate when docs comment on cost - if you're not paying for it, don't mention cost to me!
<div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote>
Let me know if you get a hold of that. I would love to learn
Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost....
<div class="FTQUOTE"><begin quote>and it being so expensive.</end quote>
He's not paying for it. Why does he care?
I hate when docs comment on cost - if you're not paying for it, don't mention cost to me!
<div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote>
Let me know if you get a hold of that. I would love to learn
saveferris2009
New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Kristen</b></i> but he was concerned about it not being approved for my mutation (yet) </end quote>
Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost....
<div class="FTQUOTE"><begin quote>and it being so expensive.</end quote>
He's not paying for it. Why does he care?
I hate when docs comment on cost - if you're not paying for it, don't mention cost to me!
<div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote>
Let me know if you get a hold of that. I would love to learn
Concerned for safety? Or efficacy? If it's efficacy, how is that a concern? if you use it, and it doesn't work, nothing lost....
<div class="FTQUOTE"><begin quote>and it being so expensive.</end quote>
He's not paying for it. Why does he care?
I hate when docs comment on cost - if you're not paying for it, don't mention cost to me!
<div class="FTQUOTE"><begin quote>He said he will at least call Vertex to see if he can get info on the in vitro results for my mutation.)</end quote>
Let me know if you get a hold of that. I would love to learn