Both CEO Chris Garabedian and CMO Ed Kaye participated. They came out fighting, for the first time addressing the slanders made against their science by an unfortunately influential blogger. They detailed for the investing community the extremely sensitive measures they have used to measure dystrophin in biopsies. They defended immunofluorescence as the gold standard and dismissed the Western Blot as outdated and less sensitive.
Ed Kaye is particularly impressive. The man is a giant in the field.
They also discussed manufacturing progress (still good), the FDA (unprecedented interest by the highest officers) and mentioned that not one eteplirsen patient has declined more than 3% from baseline.
Dirk Haussecker. He's a bitter Tekmira investor (Tekmira competed with Sarepta on Marburg and Ebola), who assumed eteplirsen wouldn't work and has spent the past year spreading misinformation about Sarepta. He has some experience with Western Blots, which enables him to fool readers without technical backgrounds. Sarepta's dystrophin paper, of course, was accepted to the most prestigious peer-reviewed journal in the field, but Haussecker, basing his criticisms solely on the edited down photos of a Western Blot in Sarepta's investor presentation, thinks he knows better. It was great to hear his ridiculous attacks get smacked down.