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 ...

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 ...

Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026Selected Development Candidates ...

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

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 ...

Neurogene received FDA Breakthrough Therapy Designation for NGN-401 in Rett Syndrome, driving a >25% share price surge. Click ...

An expert discusses the current treatment landscape of Rett syndrome including the goals of treatment and implications for utilization management strategies with the disease. David Lieberman, MD, PhD: ...

What is Rett syndrome and why does it affect only girls? Rett syndrome is a rare neurological disorder that causes victims to regress intellectually, gradually robbing their ability to think and move.

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 ...