Marisa Wexler, MS,  senior science writer—

Long-term treatment with Daybue (trofinetide) led to continual improvements among children and young adults with Rett syndrome in extension studies, with patients experiencing notable benefits in communication and other relevant issues, according to new data. Slightly more than a year ago, Daybue became the first treatment for Rett…

When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support are the biggest factors in their decision, a recent study reports. Many teenagers with Rett syndrome have difficulty breathing, and clinicians for some may recommend invasive ventilation via a tracheostomy…

The U.S. Food and Drug Administration (FDA) has given a regenerative medicine advanced therapy (RMAT) designation to TSHA-102, an experimental gene therapy for Rett syndrome. RMAT is given to regenerative medicines — cell therapies, therapeutic tissues, human cell and tissue products, or any combination using such therapies or products…

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…