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…
News
A Phase 1/2 clinical trial has been cleared to infuse NGN-401, a one-time investigational gene therapy, to a third girl with Rett syndrome, its developer, Neurogene, reports. The green light given by the Data Safety Monitoring Board overseeing the clinical trial’s progress was based on data indicating…
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…
Two young girls with Rett syndrome have been dosed with Neurogene‘s gene therapy candidate NGN-401 in a Phase 1/2 clinical trial, with data so far indicating that the treatment has been well tolerated. Another patient is expected to be dosed in the first months of 2024, with interim…
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…
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