The right SRPT deal might make VRTX management look like geniuses!
My model is $440 million revenue on 1463 US patients. Next 4 exons will bring many patients, US, Europe, Japan. Think 11,000 at 60% penetration. If GSK/Prosensa continue to have toxicity issues they will come under pressure and allow Eteplirsen in EU and Japan for a royalty.
Many more possibilities in the exon skipping world to come.