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Fostering collaborations with centers of excellence nationally and internationally.

 

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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

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Pre-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

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Rare Research Report: October 2024

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

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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.”

Pompe Disease Over Time: What We're Learning from a Natural History Study