Rett syndrome is a rare developmental disorder, most often due to a mutation in the MECP2 gene on the X chromosome, but it’s not usually inherited. Advances in genetics have launched a new era in ...

Paige Nues provides her closing thoughts regarding the treatment management of Rett syndrome. Paige Nues: We have never felt more optimistic than [at] any time in the Rett syndrome’s history in terms ...

Gene therapy may offer hope for people with a rare childhood disease called Rett syndrome. Around one in 10,000 girls are born each year with Rett syndrome, a rare genetic condition that impacts brain ...

Daybue was evaluated in a randomized, double-blind, placebo-controlled, 12-week study. Researchers at the University of Alabama at Birmingham enrolled 12 study subjects in the multi-site trial.

-- First and only approved therapy for Rett syndrome, a rare, neurodevelopmental disorder, which affects 6,000 to 9,000 patients in the U.S. 1 SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc.

Acadia Pharmaceuticals announced last Friday that the FDA approved DAYBUE (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. DAYBUE is the ...

Rebecca Day works with the charity Rett UK. She has received funding from the Economic and Social Research Council's Welsh Graduate School for the Social Sciences. Eirini Sanoudaki and Sarah Cooper do ...