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Message: Re: orphan diseases
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Jun 30, 2018 10:57PM

Hi Jupiter, 

Great question and welcome to the Agoracom hub. Unfortunately, I have limited knowledge in this area of a drug approved for one condition being available for off label use. In my opinion, a medical doctor would first go with prescribing a therapy proven effective in clinical trials for a particular condition before prescribing off label use of apabetalone. However, whether any rules exist to prevent off label prescription of drugs I do not know. I'm sure it happens. The safest route to developing apabetalone for a new condition and proving efficacy is to run a clinical trial for that specific condition. But there are so many diseases with an inflammatory component, it would be impossible to run a clinical trial for all of them.

Keep in mind that apabetalone may not have the best tissue distribution to elicit the anti inflammatory effects in the required tissues in other inflammatory conditions. Resverlogix and Zenith likely have other BET inhibitors that have a more robust anti inflammatory effects and with a different tissue distribution profile better suited for other diseases. Unfortunately, these haven't even started in Phase 1 yet, so we are years away from seeing these prove themselves in clinical trials.

Maybe someone with more knowledge in this area can better address your questions. Happy Canada Day and Independence Day to all. 

BearDownAZ 

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