Our Goal

Rare diseases come with many challenges. Some diseases are not well characterized or defined, recruitment for trials can be difficult, high-quality evidence to guide treatments is scarce, and very few expert centers are available for diagnosis, management, and research.

Our goal is to facilitate clinical research by creating rare diseases research groups to focus on related diseases, sharing the costs of our research infrastructures across the network, establish uniform studies for data collection, and make meaningful large-scale studies possible. We also directly engage with patients and their advocates, and train new investigators in rare diseases research.