Yale researchers identified an underactive mTOR pathway as a cause of lissencephaly and reversed brain malformations in patient-derived organoids using a drug. This breakthrough may offer a treatment ...

Lissencephaly is a spectrum of rare, genetic disorders in which the brain fails to develop its hallmark folds. The disorders are often associated with seizures and intellectual disability and ...

Researchers have identified a molecular mechanism driving the structural abnormalities leading to lissencephaly that have been elusive.

Tegretol and Lamictal are antiepileptic drugs that also treat other conditions. Learn more about the differences and similarities between the two drugs.

Cannabidiol is under clinical development by Jazz Pharmaceuticals and currently in Phase II for Rett Syndrome.

Leah spends her days playing with toys, painting, and molding clay, and her best friends include Noelle the Elf on the Shelf, Randall the Reindeer, and her dog, Harper. Her conditions mean Leah will ...

A six-day inquest at Norfolk Coroner’s Court heard about Holly Goodchild’s life and the events leading up to her death at the ...

When a young New York boy was diagnosed with a rare genetic disorder, hope came in an unlikely form — a golden retriever ...