Marisa Wexler, MS,  senior science writer—

The Phase 1/2 REVEAL study, which is testing the investigational gene therapy TSHA-102 in women with Rett syndrome, is expected to dose its first participant in the next month or two, according to Taysha Gene Therapies, its developer. “Screening is completed, and dosing is now scheduled for our first…

Daybue (trofinetide) is now available in the U.S. to treat Rett syndrome in patients 2 and older, according to the therapy’s developer Acadia Pharmaceuticals. The announcement comes about a month after Daybue became the first therapy to be approved for Rett syndrome by the U.S.

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…

Mice with Rett syndrome show abnormalities in their brain circuitry that helps to control movement, which may contribute to motor dysfunction that often arises as a symptom of the disease, a new study reports. The results provide “novel in vivo [in living animals] evidence for abnormal motor circuit function”…

Analyzing babies’ vocalizations using machine learning could aid in the earlier diagnosis of Rett syndrome, a small study illustrates. Researchers say such vocal analysis among infants ages 6 to 11 months may help to identify signs of Rett, or fragile X syndrome, long before a child would typically…

The U.S. Food and Drug Administration (FDA) has set an action date of March 12, 2023 to decide whether or not to approve the experimental oral therapy trofinetide for Rett syndrome. If approval is granted, trofinetide would become the first treatment for Rett ever approved by the FDA.

Levels of a small RNA molecule called miR-101a may be elevated in certain brain regions of people with Rett syndrome, according to a preclinical study. The data suggest that higher miR-101a levels can alter signals sent by nerve cells, which may impair normal brain circuitry to contribute to the…

A recent meal or snack does not affect the pharmacological profile of trofinetide, an experimental oral treatment for Rett syndrome, according to findings from a Phase 1 study in healthy adults. Food can affect how a drug moves through a patient’s body, or its pharmacological profile, the researchers…

Unravel Biosciences, which is working toward a first clinical trial to test its investigational therapy RVL001 in people with Rett syndrome, announced that it has partnered with the rare disease health platform TMA Precision Health to identify eligible patients for recruitment into the study.