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Ataluren - phase III

Hi ! Does any boy attend Ataluren Trial phase III ? I heard they just started in Cincinnati.

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Permalink Reply by Donna on August 23, 2013 at 11:28am

My son has been in the Ataluren since the beginning and continues on it. He's doing very well.

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Permalink Reply by Trinh Nguyen on August 23, 2013 at 9:12pm

Hi Donna,

Thanks for sharing. Glad that your son is on Ataluren trial and doing well. My son was just asked to join the trial in UCLA. The screen test will be done in early Oct. '13, then he shall need medical treatment per every 8 weeks if he qualifies the test. My most concern is this is double blind phase ; my son may / may not take the real medicine. It's quite tough for us and we need to travel from Viet Nam to LA at least 6 times in one year (we are living in Viet Nam).

Can you give us any advice ?

Tks/Trinh

Donna said:

My son has been in the Ataluren since the beginning and continues on it. He's doing very well.

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Permalink Reply by Blazena Kaderabkova on August 26, 2013 at 5:16pm

Hi Trinh,

we were last week in our hospital in Motol Prague and there is expected the start of the trial till two month, so we are very looking forward the trial :)

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Permalink Reply by Trinh Nguyen on August 27, 2013 at 1:01am

Hi Blazena,

It's so good that we have some chance to do something for our boy. Good luck !

Trinh

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Permalink Reply by Tracey Hartz on September 3, 2013 at 8:11pm

We are hoping to get into the Chicago site...hate double blind too, but hopefully at the end of the trial our boys will be able to take the Ataluren until it gets approved...

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Permalink Reply by Trinh Nguyen on September 4, 2013 at 5:23am

Hi Tracey. Yes, we keep hoping and praying.

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Permalink Reply by Kenny Chu on September 10, 2014 at 8:28pm

Glad to hear that. How effective is this medicine? Ataluren has been conditionally available in Europe, who knows the price?

Donna said:

My son has been in the Ataluren since the beginning and continues on it. He's doing very well.

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Permalink Reply by Trinh Nguyen on September 10, 2014 at 9:43pm

Hi Kenny!

You can check this link to update : http://ir.ptcbio.com/releasedetail.cfm?ReleaseID=848453

Though it was conditionally approved, it's not in the market yet.

Hi Donna!

Can you tell more about your son's improvement ? My son's in phase III. I feel he's in placebo because he's declining though, he's 10 now, still walks but gets more difficult in standing up. Much thanks.

Trinh