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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.
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.”
Former LDN Fellow Paves the Way for Earlier Diagnosis in Metachromatic Leukodystrophy
Laura Adang, MD, PhD, MSTR, shares how she became interested in leukodystrophy research.
