But the whole combo Sarepta FDA advisory thing is becoming a bit...

But the whole combo Sarepta FDA advisory thing is becoming a bit of a dmd soap opera. How hard would we kick up if the FDA put Sarepta on hold and asked for more clinical evidence?@RYNZN

The FDA announced a couple of weeks ago that an Advisory Committee Meeting will be held for SRP-9001 even though originally it wasn't going to be required. So the question is why the change of heart and what does it mean for the gene therapy progressed by Sarepta!!

We know that Sarepta has relied upon the use of a surrogate endpoint i.e. the production of dystrophin. Sarepta in their announcement of 16 Mar 2023 stated that its their understanding that the Advisory Committee will look at the totality of evidence to support the conclusion that the production of SRP 9001 dystrophin is reasonably likely to predict clinical benefit.

And back we come to the supposition posed by RYNZN and the DMD combination animal study announced by ANP back in February with further analysis due as early as this week. Yes, the combination study was with an exon skipping drug and not SRP-9001 but it appears likely that all synthetic dystrophin produces inflammation.