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06/20/21 1:11 AM

#315947 RE: Steady_T #315946

So I believe 2-73 does work in reducing epilepsy frequency in some girls. AVXL Needs to process data and report. You said, "So far the preliminary evidence is that it improves other aspects of a Rett girls life. That is good enough". I agree. imo. I am not a doctor.

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Investor2014

06/20/21 6:04 AM

#315958 RE: Steady_T #315946

Ladenburg Thalmann Healthcare Symposia on Rett Syndrome

From 07:00 Dr. Jeff Nuel briefly addresses seizures in Rett, which with age develop in over 80% of patients.

In the Q&A section at 27:40 Dr. Hagerman addresses seizures saying that "I did see a couple of individuals who had fewer seizures. There were some individuals who had no seizures in this study and that didn't change. I did not see anybody that had worsening of their seizures".

At about 30:00 Dr. Nuel tips in saying that "seizure burden drops as people age, seizures occur in many many people but the burden actually drops".

Now given the comments discusses are presumably based on the adult US Rett trial, I would not read too much into the seizures aspect.

Seizures, also discussed in the symposium video, are much more burdensome at a younger age but occur in fewer patients. So we have to see if the 12 week duration of the paediatric trial provides solid data on seizures.

In the only 7 weeks US Rett and the AU AVATAR studies, seizures is under other outcome measures.

Study of ANAVEX2-73 in Patients With Rett Syndrome

Seizure Frequency via seizure diary [ Time Frame: 7 weeks ]
Seizure Frequency via seizure diary


Whereas, in the 12 week paediatric Rett study seizures is a secondary outcome measure - so more important.

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome (EXCELLENCE)

Seizure Frequency via seizure diary [ Time Frame: 12 weeks ]
Seizure Frequency via seizure diary