Ataluren
Do you know anything about ataluren and their next steps?
Replies to This Discussion
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Permalink Reply by Melanie Sunny on
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I contacted to Ms. Diane Goetz last week and was told they are finalizing the data on ataluren and determining the path forward for approval and development. - Melanie
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Permalink Reply by Blazena Kaderabkova on
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Thanks, do you think already approval for treatment BDMD or other development.( Apologizing my English)
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Permalink Reply by Tamara Walters on
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Here is the latest info that was presented at a meeting in Italy this summer:
http://www.ptcbio.com/May_DBMD_Trial_Update.htm
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Does anybody know if Genzyme is working on commercialization of ATALUREN? What will be after the clinical study III for the USA patients? This study will take 36 week. What then? I have suspicion that ataluren will be available only in clinical trials.
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Permalink Reply by Sara Savalli on
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is utah the only site recruiting for this #3 study so far? out of the 37 sites listed on the clinictrials.gov site in US are most of them participating just not starting yet, and if any of them are recruiting anyone know what one's?
Blazena Kaderabkova said:Does anybody know if Genzyme is working on commercialization of ATALUREN? What will be after the clinical study III for the USA patients? This study will take 36 week. What then? I have suspicion that ataluren will be available only in clinical trials.
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Permalink Reply by Jitka Reineltová on
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In Europe, not yet available, the next steps will be decided later this year. It told me Peter Seidl, a representative for Eastern Europe. There will be so far for cystic fibrosis, where it works better.
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Yes, Utah is the only one site, but I read that Stuart Pelz told in November at Action Duchenne conference that they plan negotiations about commercialization with regulators on July this year. This is great information. But I am very worry what we can expect in EU if something.....
Sara Savalli said:is utah the only site recruiting for this #3 study so far? out of the 37 sites listed on the clinictrials.gov site in US are most of them participating just not starting yet, and if any of them are recruiting anyone know what one's?
Blazena Kaderabkova said:Does anybody know if Genzyme is working on commercialization of ATALUREN? What will be after the clinical study III for the USA patients? This study will take 36 week. What then? I have suspicion that ataluren will be available only in clinical trials.
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We were in the clinical trials and we are waiting to go back on the drug. Anyone else already started back on ?
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Where are you from?
Tamara Walters said:We were in the clinical trials and we are waiting to go back on the drug. Anyone else already started back on ?
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Permalink Reply by Ofelia Marin on
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Utah started the boys back on Ataluren for a while now.
Tamara Walters said:We were in the clinical trials and we are waiting to go back on the drug. Anyone else already started back on ?
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if you go to the clinicaltrials.gov site and look at the terminated ataluren trial it has a list of sites that did the trial, i am thinking most of the united states one's are doing this trial, except for cincinnati, but utah is the only one up and running according to the site,
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Permalink Reply by Angela Bourgeois on
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We were in the trial Boston and our son is not on it yet. We were expecting to hear something at the end of January but no word yet. Is anyone on it in Boston yet?
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