Why do you keep pressing this issue - the Etep confirmatory trial is going to start in late 2013 or early 2014 and the drugs for the other exons won't even be ready for clinical testing until later in 2014 and will require some level of safety testing before they are allowed into a larger scale trial. Give it a rest already!
See you are talking about clinical testing''does this sound better to you.
'' we are talking about the same gene and exons of that gene ,not any other gene.i could see separate trials per gene but not exons of that gene with the same drug just a sequence change..sure it is late for any to be added to the current trial maybe .future trials could be done this way several exon groups in the same trial ...
That would be nice, but it doesn't work that way, and to the best of my knowledge, there is nothing in the FDA guidelines to allow for this currently. I do believe, however, that once dystrophin is marked as a surrogate and eteplirsen successfully completes its Phase 3 trial, we could see AA given after Phase 1 trials for for each subsequent exon.
FDA's Dr. Temple, in his June talk with PPMD group talked about using surrogate marker to, basically, approve the other exons (I'm paraphrasing of course, and all imho). I thought he sounded rather enthusiastic about it too.