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PTC 124 TRIAL

Is there anyone who has gone through the trial screening or even potentially getting the drug in the 2b trial,if so how long have you been in the trial and please share your thoughts.It would be helpful for us and many more who are waiting to get in the trial.

Thankyou

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Permalink Reply by neeraj on October 10, 2008 at 4:21am

Hi All,

My son gets screened this month and hearing from parents of the 2a extension it all seems good that the drug seems to do what its ment for,Hiding the pre-mature signal.So we are hopeful and look forward for a great year ending and a start of 2009.My hope is that it will treat our boys and give them life-time therapy so be it on drug for rest of their lives but near normal life.So they can do things like normal people almost,go to university,work and etc. Is too much to expect.

BEST WISHES
Neeraj

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Permalink Reply by Melissa Yurik on October 20, 2008 at 7:41pm

My 7 year old son started in the trial about 18 weeks ago. He's tolerating the trial well. We have found that using a very small wisk mixes the medicine very well. We're not sure if he's on the medicine or the placebo but the good news is he can start taking the medicine immediately after he completes the 48 weeks of the study.

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Permalink Reply by Donnie and Linda Owens-Jacob Owens on October 28, 2008 at 7:20pm

Our grandson Jacob has been on the ptc124/placebo for 9 weeks now. We feel the same, either way after the study he will start the medicine, if he isn't already on it. We have noticed there's been a significant improvement in this verbal communication and he isn't falling as much. This can be attributed to school starting back at the same time he started the study so we're not getting our hopes up too much that's he's actually on the meds. We continue to pray for strength for what ever happens.

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Permalink Reply by Melissa Yurik on October 29, 2008 at 5:11pm

Best of luck to you!

Donnie and Linda Owens-Jacob Owens said:

Our grandson Jacob has been on the ptc124/placebo for 9 weeks now. We feel the same, either way after the study he will start the medicine, if he isn't already on it. We have noticed there's been a significant improvement in this verbal communication and he isn't falling as much. This can be attributed to school starting back at the same time he started the study so we're not getting our hopes up too much that's he's actually on the meds. We continue to pray for strength for what ever happens.