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.

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Comment by Ofelia Marin on October 15, 2008 at 4:38pm
Please let us know about the advances and hurdles that still remain.

Also, if you could ask the AVI people or their collaborators a question about their new class of drugs that showed good results in the heart and diaphragm: PPMOs...I would like to know if they plan to start a clinical trial with those as soon as the pre-clinical work is completed? Since they will start the systemic trial with the PMO skipping 51 soon, do they plan to compare the 2 chemistries before they decide which one moves to the next stage?

Thanks,
Ofelia
Comment by neeraj on October 15, 2008 at 4:18pm
Hi Pat,

you are just truly exceptional person and so many of us would have had no hope without years of hard work you have led.Many treatments and future pipelines would have not existed without you.So Thankyou

Regards

Neeraj

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