Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically fixing the underlying cause of CF. Ive done a ton of research on it, and I feel like theres no downside, theyre going to pay me to get better?! I am a bit freaked though (since they dont yet know the long term side effects), and I have to drive down to Chicago once a month (I live in Milwaukee). Im wondering if anyone else is taking part of this study, or took part in the first two? Or if youve heard how it worked in other CFers...? Im kinda going in blind here and I was just hoping someone else had experience, or if ANYONE had advice? Im just looking for support, i guess.
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Ataluren-the drug that "fixes" the CF gene
- Thread starter Milly
- Start date
Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically fixing the underlying cause of CF. Ive done a ton of research on it, and I feel like theres no downside, theyre going to pay me to get better?! I am a bit freaked though (since they dont yet know the long term side effects), and I have to drive down to Chicago once a month (I live in Milwaukee). Im wondering if anyone else is taking part of this study, or took part in the first two? Or if youve heard how it worked in other CFers...? Im kinda going in blind here and I was just hoping someone else had experience, or if ANYONE had advice? Im just looking for support, i guess.
Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically fixing the underlying cause of CF. Ive done a ton of research on it, and I feel like theres no downside, theyre going to pay me to get better?! I am a bit freaked though (since they dont yet know the long term side effects), and I have to drive down to Chicago once a month (I live in Milwaukee). Im wondering if anyone else is taking part of this study, or took part in the first two? Or if youve heard how it worked in other CFers...? Im kinda going in blind here and I was just hoping someone else had experience, or if ANYONE had advice? Im just looking for support, i guess.
Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically fixing the underlying cause of CF. Ive done a ton of research on it, and I feel like theres no downside, theyre going to pay me to get better?! I am a bit freaked though (since they dont yet know the long term side effects), and I have to drive down to Chicago once a month (I live in Milwaukee). Im wondering if anyone else is taking part of this study, or took part in the first two? Or if youve heard how it worked in other CFers...? Im kinda going in blind here and I was just hoping someone else had experience, or if ANYONE had advice? Im just looking for support, i guess.
Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically fixing the underlying cause of CF. Ive done a ton of research on it, and I feel like theres no downside, theyre going to pay me to get better?! I am a bit freaked though (since they dont yet know the long term side effects), and I have to drive down to Chicago once a month (I live in Milwaukee). Im wondering if anyone else is taking part of this study, or took part in the first two? Or if youve heard how it worked in other CFers...? Im kinda going in blind here and I was just hoping someone else had experience, or if ANYONE had advice? Im just looking for support, i guess.
katedids74
New member
Ataluren-the drug that
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
katedids74
New member
Ataluren-the drug that
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
katedids74
New member
Ataluren-the drug that
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
katedids74
New member
Ataluren-the drug that
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
katedids74
New member
Ataluren-the drug that
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
I am in a phase three for a similar trial and I would say do it! YOu are monitored closely and at this point they already know a lot of the side effects. You will help make this cure a reality!! Go for it!
mom2lillian
New member
Ataluren-the drug that
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
mom2lillian
New member
Ataluren-the drug that
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
mom2lillian
New member
Ataluren-the drug that
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
mom2lillian
New member
Ataluren-the drug that
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a>
Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
I think it rocks!
mom2lillian
New member
Ataluren-the drug that
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a><br />
<br />Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
<br />
<br />I think it rocks!
phase 3's are the safest because they have the data from 1 and 2 where the toxicity is studied extensively. Here is a good generic description of 1 vs 2 vs 3 <a target=_blank class=ftalternatingbarlinklarge href="http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
">http://en.wikipedia.org/wiki/Clinical_trial#Phase_I
</a><br />
<br />Also, if you dont like to drive in down town chicago there may be a place somehwere along the line between milwaukee and clinic that you can take a bus or a train ride in.
<br />
<br />I think it rocks!
Ataluren-the drug that
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
Ataluren-the drug that
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
Ataluren-the drug that
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
Ataluren-the drug that
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
We all appreciate you sacrificing your time to do this!
Ataluren-the drug that
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
<br />
<br />If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
<br />
<br />We all appreciate you sacrificing your time to do this!
I'm in a the 770 Ph. 3 trial. Definitely go through with screening and attempt to get in. If you decide it's not for you then at least you won't have any regrets about not having tried. And remember that even if you get placebo they will probably do an open label extension to all Ph. 3 participants which means you could have the drug years before everyone else. This could be truely a life saver for you! I say go for it!
<br />
<br />If driving into Chicago is a deterrent from participating like Nicole said consider other transportation options. Also, there's air and hired car if train or bus doesn't work schedule wise. Talk with the trial coordinator at the hospital you'll be participating at and see what they suggest. Good luck I hope you get drug.
<br />
<br />We all appreciate you sacrificing your time to do this!