Sparks, J., Michelassi, F., Thompson, J. L. P., Buchsbaum, R., Pires, N., DeRosa, J. T., Engelstad, K., DiMauro, S., Akman, H. O., & Hirano, M. (2024). A United States-based patient-reported adult polyglucosan body disease registry: initial results. Therapeutic Advances in Rare Disease, 5. https://doi.org/10.1177/26330040241227452
Subjects:
Glycogen Metabolism and Myoclonic Disorders
(OpenAlex Topic)
Molecular Basis of Rett Syndrome and Related Disorders
(OpenAlex Topic)
Lysosomal Storage Disorders in Human Health and Disease
(OpenAlex Topic)
Publication Type:
Article
Unique ID:
10.1177/26330040241227452
PMID:
Journal:
Publication Date:
Data Source:
OpenAlex
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