Does anyone have any information on these exons. I know exon skipping is not an option because of the complexity of these exons but that is where my knowledge ends..

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I am not 100% sure but I think if Exon 68 was skiped it would work for 63-67 deletion.

Look at FAQ on Prosensa's site

 Hopefull, when the first 2 or 3 of these Exon Skipping drugs are approved the FDA give all "single Exon skip drugs" a quicker approval.


Jason, thank you so much.  I almost felt that the only hope was upregulation of uptrophin.  After seeing the page you linked to the discussion I have come to realize that if 68 can be skipped and using upregulation of uptrophin my son my have a fighting chance.  I felt weak hearted but your help strengthend my heart and therefore I in-turn can be stronger for my beautiful son whom I love most unconditionally.




Tom Hilliker


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