Forest Ray PhD,  —

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…

The nonprofit Rett Syndrome Research Trust (RSRT) and Duke University’s Center for Health Measurement are asking Rett syndrome caregivers to help with a study aiming to adapt and validate a tool that could allow researchers to better understand patient responses to therapies being testing in clinical trials. The tool — called…

Targeting a biological pathway regulated by MECP2 — the gene mutated in most cases of Rett syndrome — normalized neural development in a study using mice, patient-derived cells, and a 3D model of the brain. The finding suggests that this pathway could become a therapeutic target in Rett syndrome. The…

An independent monitoring board has recommended that all three ongoing clinical trials of Anavex 2-73 (blarcamesine), an investigational treatment for Rett syndrome, continue without changes, based on a review of interim safety data. Anavex Life Sciences, the medication’s developer, announced the decision in a press release. Data…

People with Rett syndrome can receive the COVID-19 vaccine from Johnson & Johnson and still participate in future clinical trials of the investigational gene therapy TSHA-102, the treatment’s developer has announced. “We are aware of concerns about the adenovirus vector COVID-19 vaccine from Johnson & Johnson (J&J) and its…

A $2.1 million grant will fund a study investigating if — and how — cells called astrocytes might affect the irregular breathing associated with Rett syndrome. Such disordered breathing is associated with a higher mortality rate and scientists remain unsure of how mutations in the MECP2 gene — the…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Anavex Life Sciences has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for a patent application covering Anavex 2-73 (blarcamesine) and its related sigma-1 receptor (S1R) agonists in the treatment of Rett syndrome and other neurodevelopmental disorders. The…

As the Rett Syndrome Research Trust (RSRT) prepares to launch a global database of caregiver-provided information called The Rett Syndrome Global Registry, it is seeking families to participate in a pilot program to test its objectives. The goal in launching the registry is threefold: first, to provide a…

The National Institute for Neurological Disorders and Stroke, a division of the National Institutes of Health (NIH), awarded nearly $500,000 for a study seeking biomarkers of Rett syndrome. The $498,572 grant was awarded to DiamiR to conduct a study called “Circulating Organ-enriched microRNAs as biomarkers of…