Thursday, September 30, 2021 5:28:33 AM
The results of a US-based Phase 2 clinical trial evaluating the safety and efficacy of Anavex Life Sciences‘ investigational therapy ANAVEX®2-73 (blarcamesine) in Rett syndrome has been published, effectively establishing a human proof-of-concept that has resulted in a widespread celebration from within the rare disease advocacy community.
Rett syndrome is a rare neurodevelopmental disorder that almost exclusively affects girls. Estimated to occur in one of every 10,000 to 23,000 female births, the condition affects brain function and results in cognitive, emotional, sensory, motor, and autonomic problems. Classified as an autism spectrum disorder, Rett syndrome presents similarly to other rare autism spectrum conditions like Batten disease, Angelman syndrome, Fragile X disorders, and more.
The collective mindset of the Rare Advocacy Movement establishes “a win for one is a win for us all.” This sentiment couldn’t be more true in the case of today’s release of the results of the ANAVEX®2-73 (NCT03758924) trial in Rett syndrome. Here is why:
https://www.rareadvocacymovement.com/anavex-is-the-company-to-learn-from
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