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boi568

08/06/22 6:08 AM

#369302 RE: Investor2014 #369301

What do you think about the apparent change of a Phase 2 rather than a Phase 3 being planned for PD? Perhaps it is a question of knowing more about the complexities of the disease; perhaps the recent exome results, after study, may allow for a better designed Phase 3 to be reset.

Although Missling appears willing to gamble at times, such as with a single pivotal AD trial or his expressed intention of going straight to other, similar pivotal trials, I think in his own mind he believes there is little risk.

There is an American saying -- perhaps it is English -- that a good carpenter will measure twice in order to cut just once. That seems to be his style.

kevindenver

08/06/22 10:24 AM

#369329 RE: Investor2014 #369301

Very exciting stuff on the Rett front, how it's all filed with the FDA is still up for debate. We've learned how trial parameters are definitely important, as shown in the measurement debates with RQBQ-AUC versus change from baseline.

On the rare disease front in general, trials should be starting soon. Oh and the "mystery indication"...