Acadia Pharmaceuticals isn’t taking its recent rejection from the European Medicines Agency lying down, vowing to seek a ...

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

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of ...

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

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 ...

Researchers show two distinct MECP2 mutations in Rett syndrome elevate miRNA-126-3p, lowering ZO-1 and weakening blood-brain ...

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

According to the Mayo Clinic, Rett syndrome is a developmental disorder that affects the way the brain develops Courtesy of Shelly Balboa When Joseph and Shelly Balboa's daughter was 2 months old, the ...

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...

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 ...