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Message: Re: Breakthrough Therapy Designation Application

I also find it odd that DM has never said what sort of breathrough therapy he’s talking about.

I think the CF results will definitely interest BP, despite the small numbers,  but DM could have provided more information to help academics assess the strength of the data, eg 1. A plot of individual results to see if there were outliers. 2. Data on other factors known to affect CF (were mean blood pressure and mean age similar in treated and placebo groups, for example?). 3. Were the results correlated with changes in AP, HDL cholesterol, etc?

Perhaps they were presented in San Diego. 

The absence of any effect at one year is puzzling. It would have been reassuring to see at least a trend for benefit at one year, even if non-significant

And we still do not know whether eGFR improved, do we?  

 

 

 

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