Japanese company to start exon-53 skipping trial (morpholino)

A Japanese company has announced that it is starting a clinical trial for an exon 53 skipping morpholino. I don't have any more details beyond this notice, but would think it's highly unlikely that any out-of-country participants would be considered just based on how the Japanese regulatory system works.

(both Sarepta and Prosensa are also in the preclinical stages for an exon 53 skipping drug)

Press release:  http://www.nippon-shinyaku.co.jp/english/news/ns2013/2499


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Hi Sharon. My name is Alexandre and my son Rafael has deletion of Exons 48-52. Do you have any news about this Japanese study? Thank you.

Hi Alexandre--

We've had some difficulties tracking down a contact at this company but we are meeting with the Japanese Ministry of Health in October and this project is on my short list of things to ask about--will post an update here after that meeting.

Best Wishes,


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