Research

Could Trofinetide be the first drug to be approved specifically to treat Rett syndrome? On 7th December 2021, Neuren announced positive ‘top line’ results for from the Phase 3 trial in Rett syndrome. The drug had met both it’s endpoints demonstrating an improvement on the Rett Syndrome Behaviour Questionnaire and the Clinical Global Impression of…

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Lots of families are considering whether their daughter/son with Rett syndrome should have the booster Covid jab. Here Ana Abdala, member of our Scientific Advisory Board, gives her view which we hope you will find helpful. ‘A search of multiple databases did not return any specific studies of COVID vaccination in people who have Rett…

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“Hello! My name is Usree Bhattacharya and I am mom to a 5-year-old girl, Kalika, with Rett syndrome, and a faculty member in the College of Education at the University of Georgia (US). My training and expertise is in language and literacy socialization, and since my daughter’s diagnosis in 2018, I have been conducting ethnographic…

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Date: Tuesday, January 19Time: 1-2:30pm (EST) For the last 16 years, the NIH-funded Natural History Study for Rett Syndrome has been collecting data from over 1000 Rett families. Hear from NHS Principal Investigators, Drs. Alan Percy, Jeffrey Neul, and Eric Marsh on how your data has helped change the management of children and adults with Rett syndrome…

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We are excited to announce that Professor Huda Zoghbi and Professor Sir Adrian Bird have been awarded The Brain Prize 2020, worth 10 million Danish kroner. The Brain Prize 2020 was awarded for their fundamental and pioneering work on Rett syndrome which established the importance of epigenetic regulation in both brain development and the maintenance…

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