A new gene-targeting strategy that boosts a crucial brain protein could pave the way for the first effective treatment for ...

Researchers have identified a potential treatment strategy for Rett syndrome via alternative splicing modulation of the MECP2 gene.

A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report ...

A team of researchers at Baylor College of Medicine and the Duncan Neurological Research Institute (Duncan NRI) at Texas ...

Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and ...

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 approach to treat Rett ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Caregivers reported improvements in patients’ nonverbal ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterised by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...

Daybue was turned down for Rett syndrome, and Iloperidone for schizophrenia and bipolar I, with safety and benefit-risk concerns cited in both cases.

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 ...