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

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

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. 

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 :)

Hi Blazena,

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

Trinh

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...

Hi Tracey. Yes, we keep hoping and praying.

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. 

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 

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