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Started this discussion. Last reply by Bernardo A. Iriberri Jul 28, 2016. 36 Replies 1 Favorite
I am not seeing any posts or comments about the devastating news that the FDA has again chosen to sabotage Ataluren, by issuing a "Refusal to File" letter to PTC, which then led to the company…Continue
Started this discussion. Last reply by Peter Feb 17, 2014. 45 Replies 3 Favorites
Last night I watched the recording of the Ataluren webinar, held on January 17, 2013. Excuse me for not sounding particularly upbeat about it. I know that PTC and everyone at PPMD are doing as best…Continue
Started this discussion. Last reply by Linda Owens Jun 5, 2010. 24 Replies 0 Favorites
Started this discussion. Last reply by Bernardo A. Iriberri Mar 11, 2010. 7 Replies 0 Favorites
I have read the notes on Friday's call with PTC, which were posted yesterday on PPMD's main website, especially Sharon Hesterlee's useful comments. I've also read Pat Furlong's blog and her words…Continue
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Well for us the Ataluren low dose started after the high dosage trial and after the initial "limited" 30 day trial back in April 2006,. So, it has been a while. Essentially our son is 19 uses a power wheelchair to get to classes on campus and carry his books, He is a second semester college sophomore. Please do not get the idea he's "all normal" yes he can walk and do so for about 5 min now before he has to stop and rest, his upper body strength is limited, however things like cardiac and pulmonary are "normal" for a boy his size and age, so that's great. We got an extra few good years of mobility for him but more importantly his internals are good and holding steady.