Andrea Lobo, PhD, science writer —

Taysha Gene Therapies, which is developing TSHA-102 as a potential Rett syndrome treatment, raised $150 million in a private placement funding round, with most of the money supporting clinical testing of this gene therapy. TSHA-102 is being evaluated in the REVEAL Phase 1/2 clinical trial (NCT05606614)…

Individuals with Rett syndrome experience a “substantial” clinical and economic burden throughout their life, according to a new real-world study in the U.S. Altogether, the study found, the high prevalence of clinical symptoms, increased use of healthcare resources, considerable healthcare costs, and great reliance on medications and supportive…

The Rett Syndrome Research Trust (RSRT) has granted $1.1 million to help advance the development of the Emerald biosensor to accurately assess symptoms of Rett syndrome. This research award will contribute to the development of the biosensor, which uses artificial intelligence to assess irregular breathing, sleep disruptions, and…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Ketarx (racemic ketamine) for treating Rett Syndrome. The designation is awarded to products that show promise to prevent, diagnose, or treat rare diseases. Incentives for developers include seven years of market exclusivity after approval, potential…

The Rett Syndrome Research Trust (RSRT) is supporting ongoing studies of HRP-12975, an investigational therapy for Rett syndrome, using mouse models of the disease. HRP-12975, being developed by Herophilus, is reported to be the first small molecule therapy with the potential to reverse MECP2 gene deficiency, the…

A study on the brain’s molecular anomalies in Rett Syndrome received $100,000 in funding from the Canadian Institutes of Health Research (CIHR). The research will be conducted for a year by the team of Mojgan Rastegar, PhD, at the University of Manitoba, Canada. It’s one of six studies from…