News

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created…

Latest Trial Results of Anavex 2-73 ‘Very Promising’

Clinical improvements with Anavex 2-73 (blarcamesine) correlated with activation of the SIGMAR1 gene, which provides instructions for making the protein targeted by the investigational Rett syndrome therapy, in a Phase 2 study. The sigma-1 receptor, or SIGMAR1, has been associated with helping nerve cells adapt to experience and injury,…

Researchers Win $1.1M to Switch On Backup Copy of MECP2

The Rett Syndrome Research Trust (RSRT) has given two researchers $1.1 million to collaborate on the development of a therapy capable of switching on a healthy, backup copy of MECP2 — the gene mutated in most cases of Rett syndrome. The collaboration involves Antonio Bedalov, MD, PhD,…