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Anavex doses first patient in Phase 2 Rett Syndrome trial

Anavex Life Sciences (NASDAQ:AVXL) dosed the first patient in its Phase 2 trial of ANAVEX2-73 for the treatment of Rett syndrome.

Rett syndrome is a genetic neurodevelopmental disorder caused by mutations in the MECP2 gene, and is characterized by severe physical and intellectual impairments. The only treatment for Rett syndrome is symptomatic; there is currently no cure.

ANAVEX2-73, which has received FDA orphan drug designation, is an orally-administered liquid designed to activate the sigma-1 receptor protein, which is involved in restoring homeostasis.

“We have made great effort to develop a patient-centric clinical trial, using our learnings from broad dialogue with the Rett syndrome community,” Christopher Missling, Anavex’s president and CEO, said in a statement.

The trial will enroll some 30 patients 18 years of age and older to receive ANAVEX2-73 once daily, or placebo, for up to seven weeks.

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