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Prosensa has expanded its portfolio of potential treatments for Duchenne muscular dystrophy (DMD)

Prosensa has expanded its portfolio of potential treatments for Duchenne muscular dystrophy (DMD) based on skipping specific exons (sections) of the dystrophin gene; some of its exon-skipping program is being carried out in partnership with GlaxoSmithKline (GSK).
Compounds targeting exons 51 (drisapersen) and 44 (PRO044) are in clinical trials for DMD.
Compounds targeting exons 52 and 55 will be moved to clinical trials as soon as possible.
Compounds targeting exons 45 and 53 have received orphan drug designation from the European Commission and are expected to enter clinical development within the next six months.

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Permalink Reply by Phil on October 29, 2012 at 1:06pm

Surely this means the results for 51 & 44 are very positive?

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Permalink Reply by David on October 30, 2012 at 12:39am

Race is on!

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Permalink Reply by David on November 2, 2012 at 1:10am

Lisa-

Almost never do I find anything of value on the Yahoo finance message boards.

But here is a nugget that may provide some insight from business POV on what is coming out of GSK. I DO NOT PERSONALLY VOUCH FOR ANY OF THESE STATEMENTS... just thought I would share what may be the first useful exchange of ideas I've seen on Yahoo in many months

http://finance.yahoo.com/mbview/threadview/?&bn=8725fb77-7f25-3...

I too hope that the big player GSK just gives up the legal struggle and somehow licenses/buys/merges technology from Sarepta. Would save huge on legal fees alone, and the boys are biggest winners.

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Permalink Reply by Tapio on November 2, 2012 at 11:10am

Thanks for the link David. There's speculation on the message board that Sarepta's compound could be on the market by next summer if they receive accelerated approval. That seems wildly optimistic. Thoughts?