One of the pillars of gene therapy was its universality. As you...

One of the pillars of gene therapy was its universality. As you have indicated it looks like that particular pillar has been effectively discarded by Pfizer and Sarepta - excerpt from post 16 October 2021

I listened to the recent Sarepta webinar presented to PPMD titled SRP-9001 Micro Dystrophin Gene Therapy Clinical Update. The webinar while not riveting entertainment is interesting in that one gets an overview of their gene therapy program past, present and future. Specifically, Sarepta discusses the open label Study 101, the placebo controlled Study 102 in ambulatory children ages 4-7 and provides an update on the proposed EMBARK study.

The webinar goes for 70 minutes, however, the elephant in the room does not emerge until the 64^th minute during question time. Okay, I'm building up the picture. Ask yourself the question what would families impacted by DMD want most to know about from a clinical trial? Yes, safety but what else?

Referencing earlier studies but in particular Study 102, the presenters were asked "what has been seen from an efficacy standpoint so far from other studies"?

We learned a great deal more from the webinar and it was my intention to to add a further observation in regards to the discarded pillar of gene therapy but I'm currently fixated on the elephant.