Vanessa Pataia,  —

US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal

U.S. enrollment for a Phase 2 clinical trial of investigational therapy Anavex 2-73 (blarcamesine) for Rett syndrome has surpassed its initial target number of participants by nearly 50%. Anavex Life Sciences announced in a press…

Scientists Who Discovered MECP2 Link with Rett Syndrome Awarded 2020 Brain Prize

The scientists who discovered the MECP2 gene and its role in Rett syndrome have been awarded the 2020 Brain Prize, the world’s largest neuroscience prize granted by the Lundbeck Foundation. Sir Adrian…

Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will seek to advance research programs in Rett Syndrome and several other rare diseases. Besides Rett, the Center will focus on Angelman Syndrome, muscular dystrophies and lysosomal storage disorders, such as Pompe and…