News

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, and…

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, a…

Nocturnal Teeth Grinding Linked to Frequent Snoring

Bruxism, also known as teeth grinding and jaw clenching, during the night is associated significantly with frequent snoring in girls and women with Rett syndrome, an international database study shows. The data also support the previously reported high prevalence of bruxism (reported in 66.2% of patients) in this…

MeCP2-boosting Approaches Led to Toxic Events in Mice

Providing a working version of MECP2 — the gene mutated in most cases of Rett syndrome — to cells reversed disease-specific symptoms in a mouse model of a common, milder form of Rett syndrome, a study shows. However, female mice treated with this approach showed signs of excessive, toxic levels of MeCP2, the…