My grandson, 9, has finished the PTC124 2b trial, and started the extension study. He showed no improvement (got worse) during the 2b, so we were pretty sure he was on the placebo. Now, he has started the real drug 3 weeks ago. No improvement, no side effects - nothing at all. How long does it take? Should we be worrying that this is not going to do him any good? Some of you have had experience with the real drug, please share info. He is getting discouraged.

Cathy

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Hi Cathy.

I will add my general input that as far as I know drug takes some weeks or even few months(3-6) to starts working.So please do not be disappointed and remain hopeful that you will see the difference.My son is in the same trial and starts the extension trial in october so we wait until then to know for sure that he is getting the ptc124.Our hopes are positive and we would like to see all the boys benefit from this.Good luck.

Regards

Neeraj
Thanks, Neeraj. That makes me feel a little better. What have you observed in your son during the trial? Any kind of changes at all?

Cathy
neeraj said:
Hi Cathy.

I will add my general input that as far as I know drug takes some weeks or even few months(3-6) to starts working.So please do not be disappointed and remain hopeful that you will see the difference.My son is in the same trial and starts the extension trial in october so we wait until then to know for sure that he is getting the ptc124.Our hopes are positive and we would like to see all the boys benefit from this.Good luck.

Regards

Neeraj

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