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Re: Boopka post# 331433

Wednesday, 09/29/2021 12:13:45 PM

Wednesday, September 29, 2021 12:13:45 PM

Post# of 518830

From an investors standpoint an ultra rare disease limits the number of candidates



Perhaps with our safety proven this allows them to use historical controls for placebo and with a limited number of patients available, perhaps his thought process is that the trial process be accelerated thus if efficacy is shown early it may allow us get to market faster…maybe even simeoultaneously with pediatric Rett Syndrome.

All patients, rare or ultra rare are important in the eyes of God. Kudos to Dr Missling and his team for his ability to humble himself and work with each of these Rare disease groups designing the trials around meeting their needs. Im not sure how big pharma has approached this in the past but it is in the best interest of all to do so.
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