ATALUREN CLINICAL DATA DEMONSTRATE AN INCREASE IN DYSTROPHIN EXPRESSION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS

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This is great news but the FDA has left us in limbo on all drugs that INCREASE DYSTROPHIN EXPRESSION. The FDA has call into question all methods of measuring Dystrophin including immunofluorescent staining a fundamental research tool in medical science.

I agree with Jason. I still take a look at the news on Ataluren from time to time, but it all sounds like a bad dream. Anyone with a grain of common sense has known for almost four years that Ataluren significantly slows down the progression of DMD, which MUST be due to an increase in Dystrophin expression (unless we beleive in magic). That positive effect was shown during the trials that were abrutly cancelled in March 2010, when the boys on the low dose regimen supposedly "failed" to meet the primary endpoint by less than 1%, and it all really became outrageous when during the course of the January 2013 Webinar on the subject it was revealed that the boys in the low dose cohort had actually EXCEEDED the required goal! Now we are assisting to the very slow process of recruiting boys for the totally unnecessary Phase III trial, and the offiicial website for clinical trials says that it is "expected" that by June 2015 they may have their preliminary results put together. I am sorry to say that - as I predicted almost a year ago - our sons with stop codon mutations have wasted or are in the course of wasting at least five years because of the narrow-mindedness of certain people.

Good news, it Keeps Ataluren (aka PTC-124) in the cross hairs of the FDA; Once upon a time in 2001, we thought it would be FDA approved by 2004 - years later, we are still enthusiastic cheerleaders for Ataluren ...maybe someday soon enough data will convince the FDA, until then we advocate.

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