News

Better access to dental care and resources can improve quality of life for girls with Rett syndrome, according to their parents. Parents interviewed for a study on oral health-related quality of life (OHRQOL) also said their own motivation and empowerment to address their daughters’ oral healthcare concerns affected overall…

The U.S. Food and Drug Administration (FDA) agreed to Taysha Gene Therapies’ proposed design for the second part of a clinical trial testing TSHA-102, the company’s gene therapy for Rett syndrome, that’s expected to start this year. The study design is based on results from Part A of the…

Girls with Rett syndrome have significantly smaller brain volumes across all its regions, regardless of their age, a MRI study shows. Reductions in the size of certain brain regions were tied to the clinical severity of Rett patients, particularly in gross motor function, such as sitting and walking. Still,…

Taysha Gene Therapies has reached an agreement with the U.S. Food and Drug Administration (FDA) on the design and next steps to start the pivotal Part B of its ongoing Phase 1/2 REVEAL trial program, which is evaluating TSHA-102 in children, adolescents, and adults with Rett syndrome.

Bone mineral density, an indicator of bone strength, is significantly lower in girls and young women with Rett syndrome compared with healthy girls and women of the same age, a study shows. Reduced bone mineral density was tied to small body size, impaired mobility, and the use of the…

Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome. The company has submitted an application seeking regulatory clearance…

NLX-101 improves breathing and cognitive function in a female mouse model of Rett syndrome, a study found. The potential therapy activates the receptors that bind serotonin, a signaling molecule found at low levels in the brains of Rett patients. “This work provides compelling evidence of the therapeutic potential of…

Early changes in speech, including fewer varied sounds, may signal Rett syndrome before obvious developmental delays and this may help doctors reach a diagnosis sooner, a study of twin girls who didn’t share the same genetic makeup suggests. One twin developed normally, but the other, who was later…

A new study highlights how MeCP2 protein dysfunction, particularly related to changes in alternative splicing — a process by which different proteins can be made from the same gene — implicated in brain function, may contribute to Rett syndrome. Specifically, the researchers found that in both Rett patients and…

NGN-401, a gene therapy candidate for Rett syndrome that’s now being tested in a Phase 1/2 clinical trial, was demonstrated to ease symptoms and improve survival in a mouse model of the disease, per new study data. The Neurogene therapy, which uses the company’s proprietary EXACT technology,…