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 ...

A patient has died after receiving the high dose of Neurogene’s Rett syndrome gene therapy candidate. The biotech had disclosed the adverse event Nov. 11, and explained the patient was in critical ...

A new UC Davis MIND Institute study offers critical insights into Rett syndrome, a rare genetic condition that affects mostly girls. The research reveals how this condition affects males and females ...

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 ...

Paige Nues of the International Rett Syndrome Foundation explores the broader effect on a patient’s family members and caregivers. Paige Nues: To love somebody with Rett syndrome as a parent, as a ...

Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy works on reactivating healthy but silent genes ...

HOUSTON (March 4, 2026) – A team of researchers at Texas Children’s Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new ...

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 ...

Rett syndrome is a rare neurological condition that disrupts physical and linguistic development, affecting around one in 10,000 women and even fewer men. For decades, researchers assumed that people ...

-- 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.

NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, ...