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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…
From starting a Facebook fundraiser to handing out personalized information cards about Rett syndrome, supporters worldwide are marking Rett Syndrome Awareness Month, which occurs each October. Now, the International Rett Syndrome Foundation (IRSF) is offering up a host of ways people can “Go All In This October” to…
Anavex Life Sciences has been granted another U.S. patent for its lead investigational oral therapy Anavex 2-73 (blarcamesine), being developed to treat neurodevelopmental and neurological disorders, including Rett syndrome, that are driven by mutations in the MECP2 gene. This latest of a series of patents to Anavex is…
Analyzing babies’ vocalizations using machine learning could aid in the earlier diagnosis of Rett syndrome, a small study illustrates. Researchers say such vocal analysis among infants ages 6 to 11 months may help to identify signs of Rett, or fragile X syndrome, long before a child would typically…
The U.S. Food and Drug Administration (FDA) has set an action date of March 12, 2023 to decide whether or not to approve the experimental oral therapy trofinetide for Rett syndrome. If approval is granted, trofinetide would become the first treatment for Rett ever approved by the FDA.
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