News

The global prevalence of Rett syndrome is estimated to be 7.1 cases per 100,000 female individuals, according to the first known meta-analysis ever done to estimate the number of girls and women worldwide with the rare neurodevelopmental disorder. This finding “may facilitate planning of therapeutic trials in this disease,…

Girls and women with Rett syndrome who participated in a three-month, at-home physical therapy program with occasional remote supervision met or exceeded most therapy goals and gained in motor abilities, a study reports. “The proposed activity programs … improved gross and fine motor skills, and enhanced the daily physical…

Acadia Pharmaceuticals’ trofinetide has become the first treatment to be approved by the U.S. Food and Drug Administration (FDA) for Rett syndrome. It will be marketed in the U.S. under the brand name Daybue, the company announced. The medication…

A comprehensive 3D gait analysis found patients with Rett syndrome, ages 5-18, had a slow walking speed and shorter step length relative to girls without the rare neurodevelopmental disorder. The Rett patients also had wider step width and greater variability in gait features, as well as ataxia-like movements —…

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 given “positive” feedback to Unravel Biosciences‘ regarding a proof-of-concept study for RVL001, its investigational therapy for Rett syndrome, the company stated. Following a pre-investigational new drug (pre-IND) meeting, Unravel received a written response from the FDA providing guidance for…

The Rett Syndrome Research Trust (RSRT) was granted $500,000 to help test the Emerald biosensor’s ability to accurately measure symptoms of Rett syndrome. The donation was provided by Alba Tull, chair of the Tull Family Foundation and founder of First Light Capital Group. It will fund a…

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…

The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s investigational new drug application for NGN-401, an experimental gene therapy for Rett syndrome. This paves the way for the therapy to be tested in a planned Phase 1/2 clinical trial in girls with Rett syndrome, to begin later…