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Joseph_K

03/28/23 4:56 PM

#408758 RE: hnbadger1 #408685

Summation of hnbadger1's Rett treatments podcast. His link: https://www.ivoox.com/en/rett-syndrome-tomorrow-management-options-on-the-horizon-audios-mp3_rf_105253178_amp_1.html My summation is amateurish because of my lack of expertise, but maybe it's enough to satisfy some of you (and you can skip listening to it), or it may mention a topic covered in the podcast that will give you reason to listen.

Guest and host both highly credentialed doctors and professors. The guest seemed less scared off by Daybue's (trofinetide's) side effects than this board seems to feel. He categorized the diarrhea as mild to moderate and never life-threatening. Maybe if you work with Rett patients, those side effects don't sound so terrible compared to the symptoms of the condition itself? If he mentioned how small a subset of the Rett subjects the drug improved, I missed it; I recall it as only 13%, and even for them the effect was modest. If he didn't mention it, it seems a significant omission.

He spoke highly of blarcamesine, saying it seemed to have very broad, positive effects, talked a bit about its effects at the cellular level. I don't recall him mentioning Anavex by name though he repeatedly referred to blarcamesine by name. He sounded optimistic about the new pediatric trial, based on the adult trial results.

He sees a possibility that doctors may want to prescribe both drugs to the same patients. He said nothing about the possibility of either drug dominating the market (as this board, including me, expects of 2-73, provided it's approved).

Near the end he talked about a couple companies that are investigating gene therapy for Rett Syndrome. Apparently it's tricky because, as I understood it, the gene from one parent causes the condition, but a person needs the corresponding good gene from the other parent, and its difficult to stop the effect of the one without also impeding the other.