Just curious

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momtohazel

New member
it appears that ataluren has the benefit of helping 10% of the Cf population and is also in Phase 3 clinical trials, so i'm just wondering why no "buzz" about it like there has been about the vertex drugs? <br><br>any thoughts?<br>
 
M

momtohazel

New member
it appears that ataluren has the benefit of helping 10% of the Cf population and is also in Phase 3 clinical trials, so i'm just wondering why no "buzz" about it like there has been about the vertex drugs? <br><br>any thoughts?<br>
 
M

momtohazel

New member
it appears that ataluren has the benefit of helping 10% of the Cf population and is also in Phase 3 clinical trials, so i'm just wondering why no "buzz" about it like there has been about the vertex drugs? <br><br>any thoughts?<br>
 
S

saveferris2009

New member
i thought heard there were some serious side effects with ataluren.....

i hope i'm wrong and remembering incorrectly.

also, VX-770's Phase III clinical trial is already complete, and Vertex has submitted for FDA approval.

Ataluren we are still waiting to hear what the results of the Phase III trial are - and the drug certainly has't been submitted to the FDA yet.
 
S

saveferris2009

New member
i thought heard there were some serious side effects with ataluren.....

i hope i'm wrong and remembering incorrectly.

also, VX-770's Phase III clinical trial is already complete, and Vertex has submitted for FDA approval.

Ataluren we are still waiting to hear what the results of the Phase III trial are - and the drug certainly has't been submitted to the FDA yet.
 
S

saveferris2009

New member
i thought heard there were some serious side effects with ataluren.....

i hope i'm wrong and remembering incorrectly.

also, VX-770's Phase III clinical trial is already complete, and Vertex has submitted for FDA approval.

Ataluren we are still waiting to hear what the results of the Phase III trial are - and the drug certainly has't been submitted to the FDA yet.
 
H

hmw

New member
Here is an article referencing the results of the stage II trials, and it reports that the drug was well tolerated- 96% of those who enrolled completed the trial and here is a quote:
<br><div class="FTQUOTE"><begin quote>Safety results from the study showed that ataluren was generally well tolerated. Most adverse events were mild or moderate and compliance with study drug was greater than 96% for all patients.</end quote>
<br>Phase III has been an ongoing 48wk trial, and results will be reported in the first half of 2012. This is potentially a very exciting drug- not only could it help the 10%+/- of those with CF that have nonsense mutations, but similar percentages of those with a few other diseases could be benefited as well due to nonsense mutations causing their conditions.<br><br>eta> sorry, forgot to post the link!<br><a target="" title="" href="http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html">http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html</a><br>
 
H

hmw

New member
Here is an article referencing the results of the stage II trials, and it reports that the drug was well tolerated- 96% of those who enrolled completed the trial and here is a quote:
<br><div class="FTQUOTE"><begin quote>Safety results from the study showed that ataluren was generally well tolerated. Most adverse events were mild or moderate and compliance with study drug was greater than 96% for all patients.</end quote>
<br>Phase III has been an ongoing 48wk trial, and results will be reported in the first half of 2012. This is potentially a very exciting drug- not only could it help the 10%+/- of those with CF that have nonsense mutations, but similar percentages of those with a few other diseases could be benefited as well due to nonsense mutations causing their conditions.<br><br>eta> sorry, forgot to post the link!<br><a target="" title="" href="http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html">http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html</a><br>
 
H

hmw

New member
Here is an article referencing the results of the stage II trials, and it reports that the drug was well tolerated- 96% of those who enrolled completed the trial and here is a quote:
<br><div class="FTQUOTE"><begin quote>Safety results from the study showed that ataluren was generally well tolerated. Most adverse events were mild or moderate and compliance with study drug was greater than 96% for all patients.</end quote>
<br>Phase III has been an ongoing 48wk trial, and results will be reported in the first half of 2012. This is potentially a very exciting drug- not only could it help the 10%+/- of those with CF that have nonsense mutations, but similar percentages of those with a few other diseases could be benefited as well due to nonsense mutations causing their conditions.<br><br>eta> sorry, forgot to post the link!<br><a target="" title="" href="http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html">http://www.drugs.com/clinical_trials/ataluren-phase-2-data-nonsense-mutation-cystic-fibrosis-published-european-respiratory-journal-12073.html</a><br>
 
K

Kaethe108

Guest
Ataluren is our Big Hope! My daughter has One nonsense Mutation and we Hope to Read positive results soon.
I think somebody in this Forum Reported about kidney Problems with ataluren, but there is nothing official about this.
I was in contact with the patient's Service of the Company and they were very nice, answering all my questions.
 
K

Kaethe108

Guest
Ataluren is our Big Hope! My daughter has One nonsense Mutation and we Hope to Read positive results soon.
I think somebody in this Forum Reported about kidney Problems with ataluren, but there is nothing official about this.
I was in contact with the patient's Service of the Company and they were very nice, answering all my questions.
 
K

Kaethe108

Guest
Ataluren is our Big Hope! My daughter has One nonsense Mutation and we Hope to Read positive results soon.
I think somebody in this Forum Reported about kidney Problems with ataluren, but there is nothing official about this.
I was in contact with the patient's Service of the Company and they were very nice, answering all my questions.
 
B

beleache

New member
Wow, Exciting news ! Thanks for posting .. <img src="i/expressions/face-icon-small-smile.gif" border="0"> joni<br>
 
B

beleache

New member
Wow, Exciting news ! Thanks for posting .. <img src="i/expressions/face-icon-small-smile.gif" border="0"> joni<br>
 
B

beleache

New member
Wow, Exciting news ! Thanks for posting .. <img src="i/expressions/face-icon-small-smile.gif" border="0"> joni<br>
 
M

missT

Member
thank you for asking this question. I m currently in the trial. Phase 3 is completed and now we go to real drug. I heard that there were over 200 people enrolled in the study. I posted many times asking other PTCer's (atalurens) to pm me back. I only heard back from 3 or 4 people. I can honestly say that results have not been "woo hoo" like the vertex drug. I have heard talk about the kidney issues but until results come out we really wont know. I really wish other Atalurens would post. It seemed with VX770 the G551D people were dancing in the street.
 
M

missT

Member
thank you for asking this question. I m currently in the trial. Phase 3 is completed and now we go to real drug. I heard that there were over 200 people enrolled in the study. I posted many times asking other PTCer's (atalurens) to pm me back. I only heard back from 3 or 4 people. I can honestly say that results have not been "woo hoo" like the vertex drug. I have heard talk about the kidney issues but until results come out we really wont know. I really wish other Atalurens would post. It seemed with VX770 the G551D people were dancing in the street.
 
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