A collaborative project to tackle neurodevelopmental RASopathies
RASopathies are a group of rare genetic diseases that cause a wide range of symptoms from heart defects and growth delays to neurological and neurocognitive impairments that deeply affect patients and families. While treatments exist for physical symptoms, there are currently no therapies addressing the neurocognitive symptoms.
EURAS bridges this gap by bringing together 14 partners from 8 countries to advance a patient-driven registry to better understand disease progression, focus on disease modeling to understand disease mechanisms, develop new therapies and drug delivery technologies for the brain, and forge stronger connections between families, clinicians, and researchers.
