Researchers used tiny, magnetic spheres to deliver a gene editing toolbox into stem cells prepared from a patient’s skin sample and correct a genetic mutation known to cause Rett syndrome. When the edited stem cells were given cues to turn into neurons (nerve cells) in the lab, they looked…
News
NGN-401, Neurogene‘s experimental gene therapy for Rett syndrome, was designated a regenerative medicine advanced therapy (RMAT) by the U.S. Food and Drug Administration (FDA). Based on preliminary data from an ongoing Phase 1/2 trial (NCT05898620), which is testing the gene therapy in girls with Rett, the…
Daybue (trofinetide) last year became the first treatment for Rett syndrome to win approval from the U.S. Food and Drug Administration (FDA), and it’s currently up for approval in Canada. Now, the company that sells it is working to get the therapy approved in other markets.
Treatment with ADH-503, an experimental oral treatment for pancreatic cancer, was found to slow disease progression and prolong survival in a mouse model of Rett syndrome, according to a study that focused on how brain immune cells may play a role in Rett. “I hope this work will ‘move…
Long-term treatment with Daybue (trofinetide) led to continual improvements among children and young adults with Rett syndrome in extension studies, with patients experiencing notable benefits in communication and other relevant issues, according to new data. Slightly more than a year ago, Daybue became the first treatment for Rett…
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…
When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support are the biggest factors in their decision, a recent study reports. Many teenagers with Rett syndrome have difficulty breathing, and clinicians for some may recommend invasive ventilation via a tracheostomy…
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…
TSHA-102, an experimental gene therapy for Rett syndrome, appears to be well tolerated and may be of benefit in multiple areas, from gains in motor function and social skills to fewer seizures, regardless of the severity of a patient’s mutation, its developer, Taysha Gene Therapies, reported. These are…
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…
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