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for folks needing exon 53 skipping

Trial is on in Japan

https://upload.umin.ac.jp/cgi-open-bin/ctr/ctr.cgi?function=brows&a...

The most important thing I note is that age limit follows >= 5 years and < 18 years rule. 6MWT is not either in the primary or secondary criterion. Secondary criterion is tracking "Detection of exon53 skipped mRNA of dystrophin"

FDA needs to move beyond clinical measures.

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Permalink Reply by Ofelia Marin on June 24, 2013 at 9:18am

Agree! Sarepta already shows correlation b/w dystrophin and stability in 6 mwt, and I don't expect a very high correlation since many other things impact walking (weight etc.). Having FDA recognize dystrophin production as a surrogate endpoint would be huge success.

Also, I sure wish GSK would not hold their dystrophin data captive...releasing it would help the entire exon skipping field.