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Hi Rob, Exon skipping will only work to bring those out of frame back in-frame so if you have a deletion that's already in-frame than exon skipping will not work for you. That's the case with my son. He has a deletion of exons 45-47, which is in-frame. The fact that Nicholas has in-frame tho doesn't make me feel any better & I know I should. Nicholas still has a horrible disease that will shorten his life and make his life very difficult to live... been feeling kinda left out with all the exciting news about exon skipping tho.. BMDers are forgotten and that is very upsetting for me. Sorry for going off ... On the brighter side of things looks like Biomarin will be ready to go to trials early 2010 of course last year they said they would be ready early 2009 so who knows. I just hope things happen on schedule this time & this drug they have proves to be of some benefit to all suffers of this crappy disease. Take care
Roxanne. |
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Roxanne Mom to Nicholas (10) with BMD |
