PTC124 Who will it help
I understand they are having great results with this medication. But I hear it will only help a small percent of people suffering from Duchennes, I guess those affected with the stop codon. Does anybody know if they are working on a medication that will help people affected with a frameshift mutation?
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Permalink Reply by brad stringer on
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Kevin...YES! PTC124 addresses the 15% of boys with Premature Stop Codons, and so PPMD is committed to moving forward with research and cures for the other 85% of mutations. Project Catalyst is the initiative...http://www.parentprojectmd.org/site/PageServer?pagename=advancing_r...
read on...your friend, Brad -
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