Unravel Biosciences, Inc., ("Unravel"), a clinical stage therapeutics company established to advance drugs for complex diseases through its Predictable Medicine™ platform, today announced the first ...

With the FDA’s first approval for a treatment for Rett syndrome now under its belt, Daybue maker Acadia Pharmaceuticals has debuted its latest effort to raise awareness around the rare disease.

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene ...

Rett syndrome is a rare neurodevelopment disorder that leads to developmental regression, usually after a period of normal growth, and causes impairments in communication, motor skills, and speech. It ...

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 ...

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 ...

Scientists investigating the severe developmental disorder known as Rett syndrome have discovered a series of crucial molecular changes that occur long before symptoms appear. The findings could be ...

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 ...

Emiliana was an easygoing baby and a happy toddler who loved to smile, but her parents could tell something wasn’t right. She was late to learn to walk and when she was about 18 months old, they ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) ...