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Nerve cells derived from Rett syndrome patients showed alterations in structure, function, and network connectivity that depend on the severity of MECP2 genetic changes that cause the condition, a study revealed. Nerve cells from a rare atypical Rett patient with mild disease and preserved speech had similar core structural…
As part of its Project Seva, Vezbi Community Super App is supporting the International Rett Syndrome Foundation (IRSF) through a $500,000 equity donation and the creation of tailored microapps for the Rett syndrome community. The community-focused Super App was created to consolidate and organize all life aspects…
A new mutation has been found in the MECP2 gene — associated with most Rett syndrome cases — in a toddler with clinical features suggestive of congenital variant-like Rett syndrome, one of the most severe atypical forms of the disease. The child had hypercapnia, or high carbon dioxide levels…
A study on the brain’s molecular anomalies in Rett Syndrome received $100,000 in funding from the Canadian Institutes of Health Research (CIHR). The research will be conducted for a year by the team of Mojgan Rastegar, PhD, at the University of Manitoba, Canada. It’s one of six studies from…
Taysha Gene Therapies is recruiting women with Rett syndrome for the REVEAL Phase 1/2 study that will test its investigational gene therapy TSHA-102. This is the first trial of a gene therapy for Rett syndrome, according to the International Rett Syndrome Foundation. The open-label trial…
Rett syndrome has left Emily Shifflet in a wheelchair and unable to communicate verbally, but through her art and her expressive personality, she speaks loud and clear. Emily, 27, has a neurodevelopmental condition and that brings with it numerous challenges — long hospitalizations because of chronic lung disease, tube…
Case Western Reserve University (CWRU) is partnering with PharmaTher to develop and commercialize ketamine for the treatment of Rett Syndrome. “We are pleased to have added the Rett syndrome program to our clinical-stage product pipeline that focuses on novel uses and delivery forms of ketamine in the…
Taysha Gene Therapies is partnering with Astellas Pharma to support the development of its gene therapy programs for Rett syndrome and giant axonal neuropathy, another progressive neurodegenerative disorder. Astellas will invest $50 million to acquire 15% of Taysha and to receive an exclusive option to license Taysha’s…
Mice with Rett syndrome show abnormalities in their brain circuitry that helps to control movement, which may contribute to motor dysfunction that often arises as a symptom of the disease, a new study reports. The results provide “novel in vivo [in living animals] evidence for abnormal motor circuit function”…
A set of proteins in the cerebrospinal fluid (CSF) — the fluid that flows around the brain and spinal cord — are present in unusual amounts in Rett syndrome, according to an analysis of samples from rats, mice and a small group of Rett patients. Researchers identified two particular…
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