Sunday, June 05, 2022 9:30:45 AM
Well, I couldn’t read the entire letter; was behind a paywall.
First, this is not a research report. It’s a “Research Letter,” to appear in the American Journal of Medical Genetics. The journal received the letter on 26 February 2022, and accepted it on 30 April. So, it’s been seen by a good number of people at the journal. Significantly, the journal editors chose to publish the letter — they thought their readers should learn about blarcamesine and Fragile X.
To some good degree, this seems to validate what the letter states. If the editors thought the authors were stating or claiming questionable things, it would have never been accepted or published. Simply, blarcamesine as a treatment for Fragile X needs now to be properly tested in a clinical trial in real humans; beyond the transgenic rodents mentioned in the letter.
Once again, it’s the “mice aren’t men” thing. Blarcamesine naysayers will claim that it can have no viability as a treatment for Fragile X in humans until a proper clinical trial proves it. Fair enough. I’ll be holding (and perhaps increasing) my AVXL position. No reason whatsoever that humans with Fragile X won’t benefit as did the lab rodents. I’m entirely confident that the mouse models of Fragile X predicted how the drug will work in humans.
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