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- 2019
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe diseaseDOI: https://doi.org/10.1038/s41436-019-0527-9 Abstract: To characterize clinical characteristics and genotypes of patients in the ADVANCE study of 4000？L-scale alglucosidase alfa (NCT01526785), the largest prospective United States Pompe disease cohort to date
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