Fostering collaborations with centers of excellence nationally and internationally.
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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 translational research?
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.”
