Translating patient priorities into objective endpoints for autonomic dysfunction in AHC – RARE Hope is a patient-centered organization dedicated to accelerating the development of therapeutics for rare neurological diseases. Adopting a unique “platform approach,” the organization focuses on Alternating Hemiplegia of Childhood (AHC) as a prototype to build a scalable infrastructure for research, biomarker development, and clinical trial readiness. By leveraging common biological mechanisms and a shared scientific and clinical toolkit — and by uniting scientists, clinicians, and patient families — RARE Hope aims to incubate innovation and de-risk the therapeutic pipeline, ensuring that promising treatments can move rapidly from the lab to the patients who need them.