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georgejjl

06/19/21 11:05 AM

#315790 RE: Pazzo1212 #315787

My thoughts are that she may not be still taking ANAVEX®2-73 (blarcamesine).

Maybe they should consider giving Annelise her daily dose of ANAVEX®2-73 (blarcamesine) about an hour or so before her sleep time. They may also consider adjusting her timing and/or dose level of her AEDs.

This is NOT meant as medical advise. This is just food for thought.

Good luck and GOD bless Annelise and her loved ones.
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plexrec

06/19/21 11:11 AM

#315792 RE: Pazzo1212 #315787

Pazzo--"Seizures leave us all feeling helpless"--concerning since she has been taking 2-73--but we don't know "the whole story" !!!
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98beamer

06/19/21 11:49 AM

#315797 RE: Pazzo1212 #315787

Annelise spent last night wired up for a Video EEG in the Epilepsy monitoring unit at Westmead Children's Hospital. We have been concerned by her nocturnal seizures that have been waking her from sleep.



Pazzo1212
Could you give us some context as to Annelise and her condition before the confirmed dose of Anavex 2-73? What was the severity of her seizures and how often did they occur before the known/confirmed doses?

God bless Annelise and we pray for relief.
TIA
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hfb46

06/19/21 2:01 PM

#315824 RE: Pazzo1212 #315787

Pazzo, you stated that you are not aware if she has been receiving the drug or the placebo, and that you only know for sure that she received her first dose of the clinical trial drug at the beginning of the OLE. If she was not receiving A2-73 previously, and has just now started taking it, it is too early to draw conclusions about its efficacy for the seizures. The vast majority of medications need a little time for their MOA to effect a desired response, even with those meds that raise the blood level of the active substance quickly. Let's hope that she was NOT taking the A2-73 previously, and has just now started taking the active drug, and it will have a desired effect very soon. Good luck.
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frrol

06/19/21 2:50 PM

#315833 RE: Pazzo1212 #315787

Brace yourself for the confirmations.

But realistically, 2-73 is not necessarily going to be 100% effective, nor treat all symptoms. Anecdotal stories like this are unfortunate, and not surprising in biotech. These companies do not make magic beans. There are likely 'improvement" anecdotes, too. Our trials' full results will give us a good idea of how effective it is, in what manner, and under what conditions (eg, dosing, S1 status, age). It's why we do them. (Not because the FDA is mean and stupid and corrupt and...)

Btw I think Doc328 expressed doubts about our anti-seizure potential from the beginning, due to our hypothesized MOA. He may be right. But the company is hopeful.

Looking forward to full results!
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frrol

06/19/21 3:53 PM

#315858 RE: Pazzo1212 #315787

Also interested in dadofmarcmax's view.
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abew4me

06/19/21 5:59 PM

#315890 RE: Pazzo1212 #315787

This comes back to the 80% efficacy for those participants who have the wild type gene. If only 80% of the population has the WT Gene, that means that 20% of the population doesn't have it...and thus, will not respond as effectively.

Unfortunately, it sounds like Annelise is in the 20% group.


Please note: I am not a doctor so take my analysis with a grain of salt.
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dadofmarcmax

06/19/21 11:23 PM

#315931 RE: Pazzo1212 #315787

When people have intractable epilepsy, introduction of new antiepileptic meds can often change the seizure subtypes or worsen a particular seizure type while improving another.


Let’s not get ahead of ourselves and start thinking 2-73 cures everything.

Personally I think 2-73 may work in synergy with antiepileptic meds instead of in lieu of antiepileptic meds.

If 2-73 restores cells to their pre-distressed selves by restoring cellular homeostasis, what happens when cells/neurons themselves are diseased/dysfunctional at baseline and you use 2-73? Aren’t you just restoring these inherently hyperexcitability-prone neurons back to their normal selves with 2-73 and the Rett kids?
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Jager997

06/20/21 12:26 PM

#315992 RE: Pazzo1212 #315787

Source of this post?

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georgejjl

06/20/21 2:08 PM

#316006 RE: Pazzo1212 #315787

What we do NOT know is whether Annelise is still taking Anavex 2-73 Blarcamesine or whether she had to discontinue taking Blarcamesine for some reason.

I pray for Annelise and all the other girls in the Rett syndrome trials each and every evening.

Good luck and GOD bless,