Rett syndrome is a rare and severe, neurodevelopmental disorder primarily affecting females, caused by mutations in the MECP2 gene. It typically manifests after an initial period of normal development, with symptoms emerging between 6 to 18 months of age. These include severe loss of motor skills, communication abilities, and purposeful hand movements, often accompanied by repetitive hand stereotypies, such as hand-wringing or clapping. Cognitive impairments, breathing irregularities, and seizures are also common.

iNGENū’s team of researchers and clinicians is dedicated to advancing Rett syndrome research. Through innovative trial designs and a patient-centered approach, we work to accelerate the development of new treatments that could offer improved options and outcomes for those affected by Rett syndrome.