Exon 17 skipping
Information
Only 1.9% in the cases of exon deletion;
Only 1.9% opportunity for receiving treatment;
Let's join hands for our sons' future !
Members: 4
Latest Activity: Jan 29, 2016
Hi !
Discussions
How many people in similar case ?
Started by Nguyen Van Anh Jan 15, 2011. 0 Replies 0 Favorites
1.9% is really small but if people all join hands, future must open wide.
Comment Wall
Comment
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Comment by David on September 5, 2011 at 4:30pm
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I believe the best opportunity for our boys is to convince regulators to approve exon skipping drugs as a class. Thus, clinical trial results for exon skipping 51 or 44 would open doors for all other formulations. A long shot, to be sure, but far more likely than waiting for a pharma or biotech company to address such a small population.
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My son has deletion in exon 16 and as such would need to skip 17 and 18. Why both? Because skipping 17 only does not allow connection to 18. As shown by the exon chart for dystrophin, 16 can connect to 19 but not 18.
http://www.muscular-dystrophy.org/about_muscular_dystrophy/research...
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