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Wednesday, 12/29/2021 11:50:30 PM

Wednesday, December 29, 2021 11:50:30 PM

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Fragile X premutation review article published today and co-authored by Dr. Hagerman: https://www.dovepress.com/fragile-x-premutation-medications-therapy-and-lifestyle-advice-peer-reviewed-fulltext-article-PGPM

Another new treatment which is pending FDA approval for research in FXTAS is Anavex 2–73, a sigma 1 agonist that works in the endoplasmic reticulum and mitochondrial interface. It can reduce oxidative stress, alleviate calcium dysregulation in neurons and improve mitochondrial function which is greatly needed in FXTAS and in other disorders.127,128 Anavex 2–73 also helps other cellular dysfunctions including proteostasis, autophagy and neuroinflammation that are present in neurodegenerative and neurodevelopmental disorders.129,130 It has been efficacious in animal models and human studies in Rett syndrome (unpublished), studies in Alzheimer Disease, in Parkinson's Disease Dementia (unpublished) and most recently in rescuing hyperactivity and other behaviors in the KO FXS mouse.127,131,132 Soon controlled trials of Anavex 2–73 will be studied in those with FXTAS.



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