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 —…
News
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…
Scientists may have discovered promising treatments for Rett syndrome in two small molecules, called KW-2449 and VPA, each seen to ease nerve cell deficits — thereby potentially improving the flow of information between neurons — in a lab model of the disorder. “These findings suggest that KW-2449 and VPA…
Treatment with the diabetes medication metformin for four months led to cognitive improvements and restored brain energy production in a mouse model of Rett syndrome, a study found. Notably, the therapy’s benefits were observed when administered in a relatively advanced stage of disease — one in which the…
Girls with Rett syndrome caused by MECP2 gene mutations have increased deep sleep and reduced rapid eye movement (REM) sleep, a study shows. The sleep patterns are generally similar across the patients’ genetic and clinical, or disease severity, characteristics. However, increased hand disability was associated with a…
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