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 ...

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 ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

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 ...

Natural history data analysis established patients ≥ six years of age are in developmental plateau, with a ~0% likelihood of gaining/regaining developmental milestones across the core functional ...

--DAYBUE is the first and only therapy approved in Canada for the treatment of Rett syndrome, a rare, neurodevelopmental disorder SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) ...

Taysha Gene Therapies, Inc. announced key updates on its TSHA-102 program, an investigational gene therapy for Rett syndrome, at the upcoming 2025 International Rett Syndrome Foundation Scientific ...