Marisa Wexler, MS,  senior science writer—

The Rett Syndrome Research Trust (RSRT) is teaming up with partners in industry and academia to get ready for a study called VIBRANT, which plans to test innovative ways to measure and potentially monitor the symptoms of Rett syndrome in patients, with a focus on autonomic dysfunction. The…

The first girl with Rett syndrome has been given the experimental gene therapy TSHA-102 in a clinical trial, according to the treatment’s developer, Taysha Gene Therapies. “Dosing the first pediatric patient with Rett syndrome marks an important step forward in our efforts to broaden the clinical evaluation…

Treatment with Anavex 2-73 (blarcamesine) led to improvements in behavior among children with Rett syndrome in a Phase 2/3 clinical trial — but the extent of these gains was not significantly different from improvements seen in patients given a placebo. Those are the top-line findings of the EXCELLENCE…

Rett syndrome is marked by unusually low levels of LAT1, a protein that’s needed to move amino acids — the building blocks of proteins — into the brain. That’s according to the study, “Metabolic characterization of neurogenetic disorders involving glutamatergic neurotransmission,” published in the Journal of Inherited…

Electrically stimulating a specific region of the brain led to improvements in memory in a mouse model of Rett syndrome — and these benefits persisted for several weeks after the brain stimulation was stopped, a new study reports. Repeated brain stimulation could promote even longer-term memory improvements, according to…

Treatment with a modified version of a signaling molecule called NGF improved survival and normalized behavior in a mouse model of Rett syndrome, a new study reports. The study, “Reversal of neurological deficits by painless nerve growth factor in a mouse model of Rett syndrome,” was published…

A second adult with Rett syndrome has been given the experimental gene therapy TSHA-102 in an ongoing clinical trial, according to an announcement from the therapy’s developer, Taysha Gene Therapies. “Dosing the second adult patient in the REVEAL Phase 1/2 adult trial in Canada marks important progress…

Girls with Rett syndrome have detectable abnormalities in the electrical activity in their brains, a new study confirms. The study, “Abnormal spectral and scale-free properties of resting-state EEG in girls with Rett syndrome,” was published in Scientific Reports. Brain cells communicate with each other by sending…

Repetitive movements of rubbing the hands together are associated with an increased risk of skin injuries in people with Rett syndrome, but repetitive movements of raising the hands to the mouth are linked with better mobility in the shoulders and elbows. That’s according to the study, “…

The first woman with Rett syndrome given the gene therapy TSHA-102 has experienced improvements in her sleep, breathing, and motor skills in the first weeks since the treatment. That’s according to new data announced by Taysha Gene Therapies, the company developing TSHA-102. “We believe the initial safety profile…