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Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease GENETICS IN MEDICINE Kishnani, P. S., Gibson, J. B., Gambello, M. J., Hillman, R., Stockton, D. W., Kronn, D., Leslie, N. D., Pena, L. M., Tanpaiboon, P., Day, J. W., Wang, R. Y., Goldstein, J. L., Haack, K., Sparks, S. E., Zhao, Y., Hahn, S., Pompe ADVANCE Study Consortium 2019; 21 (11): 2543–51
View details for DOI 10.1038/s41436-019-0527-9
View details for Web of Science ID 000494989200018