Andrea Lobo, PhD, science writer —

The International Rett Syndrome Foundation (IRSF) designated three new clinics as centers of excellence, saying they provide best-in-class clinical care for people with Rett syndrome. The designation comes with funding support from the foundation and integration into IRSF’s Center of Excellence Network, which now has 21 clinics…

CNM-AU8, an experimental gold nanocrystal therapy from Clene Nanomedicine, was seen to significantly improve mitochondrial function and nerve cell health in a cell model of Rett syndrome. Preclinical findings were detailed at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado, in the…

Rett Syndrome Research Trust (RSRT) has launched an initiative to advance three genetic medicines for Rett syndrome to clinical trials by 2028. Called Roadmap to Cures, it seeks to raise $40 million over the next four years to help select and develop the therapies and is based…

NGN-401, Neurogene’s gene therapy candidate for Rett syndrome, was well tolerated in the first three patients dosed in a Phase 1/2 clinical trial. The Phase 1/2 trial (NCT05898620) is enrolling girls with Rett, ages 4-10, at three sites in the U.S. The company also plans to establish…

Children with the MECP2 gene-related disorders Rett syndrome and MECP2 duplication syndrome (MDS) experience atypical sensory processing, according to a recent study. Deficits in the MeCP2 protein chiefly cause Rett syndrome, whereas MDS occurs due to duplication of the chromosomal region containing the MECP2 gene. Unusual sensory effects…

The use of low-dose extracorporeal shock wave therapy — a safe and noninvasive treatment known as ESWT — for three months was found to improve motor function in children with Rett syndrome. Reduced muscle stiffness, known as spasticity, also was seen among the children, but the benefits were not…

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…

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…

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…

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)…