Wednesday, September 18, 2019 10:31:31 AM
Are we seemingly on track to alleviate: repetitive hand wringing, slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability?
Assuming so, might this data presented at the Rett Congress in Finland next week be highly encouraging to RS & epilepsy researchers and their patients/families?
Suppose we’ll find out on 9/27 and 9/28.
From the 2016 preclinical mouse study p/r:
https://www.anavex.com/anavex-presents-preclinical-results-of-anavex-2-73-in-rett-syndrome/
“Chronic oral daily dosing of ANAVEX 2-73 starting at ~5.5 weeks of age was conducted in the MECP2 Rett syndrome disease mouse model and continued through a 12-week behavioral testing time point 60 minutes pre-treatment during the behavioral testing. Behavioral paradigms measure different aspects of muscular coordination, balance, motor learning and muscular strengths, some of the core deficits observed in Rett syndrome.”
And,
“The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability.”
As you noted, on Monday Dr Missling indicated:
“Neurobehavioral effects of ANAVEX®2-73 (blarcamesine) previously observed in preclinical studies were also detected in patients with Rett syndrome, pointing to the ability of translation of preclinical to clinical data. ANAVEX®2-73 (blarcamesine) has received orphan drug designation from the FDA and EMA for the treatment of Rett syndrome.”
https://www.anavex.com/anavex-life-sciences-announces-preliminary-clinical-efficacy-data-of-its-u-s-phase-2-clinical-trial-of-anavex2-73-in-patients-with-rett-syndrome/
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