%0 Journal Article
%T Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease
%J -
%D 2019
%R https://doi.org/10.1038/s41436-019-0527-9
%X To characterize clinical characteristics and genotypes of patients in the ADVANCE study of 4000£żL-scale alglucosidase alfa (NCT01526785), the largest prospective United States Pompe disease cohort to date
%U https://www.nature.com/articles/s41436-019-0527-9