Description

This opportunity supports efficient and innovative natural history studies focused on rare diseases or conditions with unmet medical needs. The goal is to generate high-quality, interpretable data that address critical knowledge gaps, remove barriers to progress, and broadly impact the development of medical products for rare diseases. Projects may target a single rare disease or multiple related diseases with similar pathophysiology. The program encourages studies that will facilitate the development and evaluation of new therapies, diagnostics, or interventions in the rare disease space.