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biocqr

12/28/14 4:45 PM

#185214 RE: poorgradstudent #185212

CLDN > Is the statement below any cause for optimism?

Still, in what Zeiher called "the most important message of this pilot trial," there was evidence of a dose-response effect for two parameters that reflect ventricular remodeling—specifically, natriuretic peptide levels, which rose substantially less over six months in the SERCA2a groups than in the placebo group (they didn't rise at all in the high-dose group), and LV end-systolic volume, which rose sharply in the placebo group and declined in the high-dose SERCA2a group.



http://www.medscape.com/viewarticle/790633#vp_2

WID

12/29/14 11:55 AM

#185230 RE: poorgradstudent #185212

Poorgrad, According to you, FDA panel might make a mistake to grant CLDN the Breakthrough Designation Therapy without looking at the info you presented?

I doubt that the company mislead the FDA on the CUPID1 data or the FDA shortsighted on the info you mentioned.

xcernfsi

12/31/14 7:58 PM

#185324 RE: poorgradstudent #185212

Re: Celladon math

Thanks for this, PGS. Do you have a link to where "They note that they see anywhere from 20 to 365 copies of the AAV in a typical ug of DNA."?