ataluren what are they doing?
it seems that PTC didn't give any dates and planning for the compassionate treatment in naples
no result of the biopsy!
we can't accept to wait and wait for .. no answer
Replies to This Discussion
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Permalink Reply by Ofelia Marin on
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How do you know? Have you seen the Naples presentation? I think Pat Furlong is attending this conference.
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Permalink Reply by Melanie Sunny on
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This morning I emailed to Diane M. Goetz and asked if the presentation for ataluren in Naples will be able to be posted, and she told me that they are preparing a summary for parents to be posted by the end of next week.
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Permalink Reply by ferre on
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we could follow this discussion on my private e mail i you were on the trial
Melanie Sunny said:This morning I emailed to Diane M. Goetz and asked if the presentation for ataluren in Naples will be able to be posted, and she told me that they are preparing a summary for parents to be posted by the end of next week.
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My son is 4 years and 11 months, and he is not on the trial.He has a non-sense mutation and might benefit from ataluren. Best, Melanie
ferre said:we could follow this discussion on my private e mail i you were on the trial
Melanie Sunny said:This morning I emailed to Diane M. Goetz and asked if the presentation for ataluren in Naples will be able to be posted, and she told me that they are preparing a summary for parents to be posted by the end of next week.
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Permalink Reply by Christine Piacentino on
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I know that Pat Furlong is attending the meeting in Naples. PTC will post the summary by the end of next week. At the Conference in Denver. They promised that they would provide an update regarding next steps by the end of this month.
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Permalink Reply by tom evans on
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Ben was in the trial at Philly, I hope we hear something soon because he is getting much weaker since stopping the trial
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I also hope that they post at least a summary of their presentation in Naples, and Pat's comments for the community will be much appreciated too. This is extremely important not only for those whose children were involved in the trials but also for the hundreds of DMD boys and families who live abroad and could not participate in those trials. In my son's case Ataluren continues to be the only concrete hope for at least slowing down the course of the disease until something else comes up. I still think it is crazy that they stopped the trials in March simply because of that ONE METER (three feet) difference between the expected goal and the real outcome in terms of distance walked during the 6-minute test.
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What happened with this? Weren't we supposed to get an update from whomever attended this conference?
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Permalink Reply by BeritSofie on
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Still waiting to hear results of Ataluren from the presentation in Naples.
Thank you Bernardo for thinking of all the boys who live abroad and couldn't participate in this trial. Our boy, age 11, is one of them !!
"she told me that they are preparing a summary for parents to be posted by the end of next week".
Diane M. Goetz, posted July 21.
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Hi Berit,
My son Hernán is also one of the hundreds of boys who did not participate in the trials because we live abroad (in this case, Argentina).
I've just spotted PTC's announcement on the Naples conference, which has been uploaded to PPMD's website. It looks like the parents of boys who took part in the trials are encouraged to discuss the possibility of receiving Ataluren on an individual basis.
I am attaching a copy of that announcement.
Best wishes.
Bernardo
BeritSofie said:Still waiting to hear results of Ataluren from the presentation in Naples.
Thank you Bernardo for thinking of all the boys who live abroad and couldn't participate in this trial. Our boy, age 11, is one of them !!
"she told me that they are preparing a summary for parents to be posted by the end of next week".
Diane M. Goetz, posted July 21. - Attachments:
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hi
it seems that PTC gives the opportunity for american children who were on trial to get them ataluren. For the non american children who have also followed the trial!!!!!!!!!!!!!!!!!!!!!!!!!!! NO ANSWER!!
if somebody has information on that topic other than "see with genzyme" it interest a lot of parents!
best
françoise
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