News

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,…

The protocol of the Phase 1/2 trial (NCT05898620) that’s evaluating Neurogene’s NGN-401 in girls with Rett syndrome has been amended to use the low treatment dose of the gene therapy for all future participants, the company announced. The protocol alteration was submitted to the U.S.  Food and Drug…

One and a half years of daily Daybue (trofinetide) treatment safely improved communication, eye contact, and hand use among girls ages 2-4 with Rett syndrome, according to the final published results of the DAFFODIL trial, which tested the approved therapy in the youngest of patients. Although most patients…