This proves that the lack of trust by the FDA on dystrophin meaures is unfounded. The FDA has what it need to make a decision on Eteplirsen.
A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy
I also think this proves that the FDA has all they need Jason.
Thank you for posting the article here Jon Moulton. I had posted it on Facebook in answer to a discussion about the problems with dystrophin quantitation and the anouncement that Sarepta would now delay filing with the FDA but the discussion disappeared and no one commented on it while it was still up, as far as I had witnessed anyway.
As a scientist, what do you think about Prosensa's article Jon Moulton? Is it a sensitive, reproducible and objective method(s)? Could it be used to convince the FDA that drytrophin quantitation can be used for filing and approving new drugs for DMD now ? Or has it confused the FDA about dystrophin? Just trying to wrap my head around it all. Thanks.
Ultimately the FDA will decide what level of precision and accuracy are acceptable in an assay. I have not seen them specify levels of precision and accuracy that they would accept. Without a target, when methods are published the FDA is free to accept or reject them. If the FDA specifies what they will accept, it becomes an engineering problem to meet the specifications. Without a specification, the decision to accept or reject is political.
Yes, well it's the politics that is killing me, figuratively, and my son literally.