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MI Dendream

05/12/22 5:41 PM

#472299 RE: Doc logic #472297

DocL, Just to add, a universally fatal orphan disease where the treatment in question has no adverse effects.

As a source of relative reference please look at the Exondys 51 and note the lack of efficacy and failed trials along with a paucity of safety experience.

https://www.exondys51.com/modules/exondys/files/EXONDYS51PI.pdf

It sells for about $450k per annum if not mistaken and it does sell.