News

Injections of botulinum toxin in the muscles involved in chewing were found to reduce teeth grinding in an 8-year-old boy with Rett syndrome, according to a case report. Botulinum toxin blocks the release of acetylcholine, a chemical messenger, at the neuromuscular junction — the place where nerve cells and…

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…

October is Rett Syndrome Awareness Month, and the International Rett Syndrome Foundation (IRSF) is encouraging people to go “All In” to support patients and boost critical research. “This October during Rett Syndrome Awareness Month, there are so many ways you can join IRSF to help increase awareness of Rett syndrome…

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, “…

A lack of MECP2 protein, the chief underlying cause of Rett syndrome, prevented precursor cells from transforming into fully mature nerve cells in the early stages of brain development, a study suggests. Using 3D organoids that mimic the brain, blocking a pathway elevated by MECP2 depletion enhanced the formation…

TSHA-102, an experimental gene therapy for Rett syndrome now being tested in clinical trials, has been granted fast track status by the U.S. Food and Drug Administration (FDA). “We are pleased to receive FTD [fast track designation] from the FDA, which underscores the significant unmet medical need in patients…

Taysha Gene Therapies, which is developing TSHA-102 as a potential Rett syndrome treatment, raised $150 million in a private placement funding round, with most of the money supporting clinical testing of this gene therapy. TSHA-102 is being evaluated in the REVEAL Phase 1/2 clinical trial (NCT05606614)…

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…