I am participating in the exon skipping (aon) meeting at Banberry Center. All of the leaders in the AON field are here as well as the basic scientists invovled in this space. We are here to discuss issues related to antisense as a potential therapy for dmd. You are already aware, prosensa is currently involved in a dose escalation trial in 3 locations in Europe. The trial involves 1518 patients, skipping exon 51. It is dose escalation trial and currently recruiting patients. This trial is to evaluate safety and tolerability. The trial involves a sub-cutaneous injection 5 times/wk and increasing the dose once it is determined safe to go on. It is known that this approach cannot restore lost muscle.
I will continue to post as the meeting progresses.