Trial is on in Japan

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.

Views: 219

Reply to This

Replies to This Discussion

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.

Reply to Discussion


Need help using this community site? Visit Ning's Help Page.



© 2023   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service