News

Leriglitazone, an oral treatment candidate for neurological diseases, can improve the function of mitochondria — known as the powerhouse of the cell — in lab studies with cells from Rett syndrome patients and in a mouse model of the disease. Specifically, leriglitazone therapy restored energy production and reduced oxidative…

Effective communication is a top concern for people affected by Rett syndrome or related disorders, a U.S. caregiver survey has found. Other top concerns for patients include seizures, problems with walking or balancing, loss of function in the hands, and constipation. Recent progress in understanding brain development disorders such…

The Rett Syndrome Research Trust (RSRT) has launched a global registry for parents to share their knowledge and experiences about caring for their loved ones with Rett syndrome. Called the Rett Syndrome Global Registry, its aim is to advance the development of genetic medicines for Rett. The…

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…

To recognize the hopes and pursuits of the siblings of those with Rett syndrome — while highlighting the disease’s impact on patients’ families — Acadia Pharmaceuticals has established the Rett Sibling Scholarship program for U.S. residents. The new program will award 10 higher education scholarships of $5,000…

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…