Fostering collaborations with centers of excellence nationally and internationally.

Join One of Our Research Studies
Participants make it possible for researchers to find new treatments, speed diagnosis and improve the lives of those affected by rare diseases
Find a StudyPre-Doc Preparatory Program (P³) in Translational Research
Do you have a rare disease research coordinator, genetic counseling assistant, or technician who may benefit from formal training in clinical and t

Rare Research Report: October 2024
Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications.

Pompe Disease Over Time: What We’re Learning from a Natural History Study
To learn more about Pompe disease, the Lysosomal Disease Network (LDN) is conducting a natural history study, “Lysosomal Disease Network Longitudinal Study of Pompe Disease.”
