Jacob was on the drug high dose for over a year.  We felt like he had more mobility and energy when taking the drug. Not sure if this image or fact. Seems like since it's use was suspended by FDA it seems like Jacob almost 11 has been declining fast. 

However, this also could be his age and the natural progression.

Not sure if this is helpful or not.  Looks like the FDA will be approving the drug use again in the next 2 months or so according to Cincinnati.




Chad Studebaker

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My Jacob was also in the PTC trial but he was on the low dose. We could see an improvement in his mobility, endurance and stamina. I pray that we will get him back on it very soon as he too is getting worse.

Linda Owens
My son Kyle was also in this study. I talked to his clinical trial nurse actually this week and they are hoping that getting the boys back on the drug will be within the next six weeks - hope so!!!



Thank you for responding and sharing your observations. 


My latest understanding is that PTC is working to get all the paperwork into the FDA by the end of 2010.   Then, if my understanding is correct, the FDA then has six months to provide a 'go' or 'no go', or to demand more trials.   Assuming a 'go', PTC would need time to ramp up production.   My best guess estimate for access to Ataluren for boys who did not participate in the trials is Nov 2011.   I sure hope it is faster though!


I spoke to and met some people from PTC.  They were fantastic! 


Best to you and your family.


Bob Getler



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