“RARE Hope looks for opportunities to maximize impact by advancing shared infrastructure and tools that can support multiple conditions at once. These core digital measures for pediatric rare disease were developed to be biologically and clinically appropriate for individual conditions while fitting within a shared, scalable framework. Shaped by diverse, multidisciplinary input, they enable consistent, objective, regulator-ready assessment across disorders — delivering tools and infrastructure that can ultimately improve the lives of more patients and families.”

– Nina Frost | President, RARE Hope