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Treatment with trofinetide is tied to improvements in caregiver-reported outcomes by 9 months in individuals with Rett ...
More information: Osman Sharifi et al, Sex-specific single cell-level transcriptomic signatures of Rett syndrome disease ...
Poppy Allard was a seemingly healthy baby, when, at around 15 months, her behaviour suddenly changed - now she’s unable to ...
Formerly thought to be a neurodegenerative disease, Rett syndrome (RS) is a neurodevelopmental arrest of the brain that almost exclusively affects females and occurs in a variety of racial and ...
Top-line data from the LAVENDER study in 187 girls and young women with Rett have shown ... of patients taking the drug – and vomiting. Rett syndrome is caused by mutations in the MECP2 gene ...
Tom Farnell is raising money for Ronald McDonald House Charities, which provides accommodation near hospitals.
Rett Syndrome is a rare genetic neurological disorder ... Then Came a Surprise (Exclusive) Woman Always ‘Keeps the Peace’ with Her ‘Selfish’ Parents. Now She Wants to Finally Say Something ...
It affects about 15,000 girls and women in the US and 350,000 worldwide ... drug on one of the primary endpoints of the trial, the Rett Syndrome Behaviour Questionnaire (RSBQ), but that did ...
For parents of children with Rett syndrome – a rare neurodevelopmental disorder that makes daily life challenging – it’s the small moments that bring the most joy. Although 14-year-old ...
95.8% girls and women) prescribed trofinetide as routine clinical care for all types of Rett syndrome are tasked with providing patient assessments based on behavior, quality-of-life and ...
The Blue Bird Circle Rett Center is one of the nation’s largest resources for families of children with Rett syndrome. Our center specializes in the diagnosis, treatment and follow-up care of girls ...
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