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Enzyme replacement therapy

The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease

Esther Kuperus, Jan C. van der Meijden, [ ... ], W. W. M. Pim Pijnappel

Circulatory C-type natriuretic peptide reduces mucopolysaccharidosis-associated craniofacial hypoplasia in vivo

Marina Kashiwagi, Kazumasa Nakao, [ ... ], Kazuhisa Bessho

Exploring the diagnostic potential of miRNA signatures in the Fabry disease serum: A comparative study of automated and manual sample isolations

Josephine Y. Fang, Saravanan Ayyadurai, [ ... ], Mark G. Qian

Association between under-dose of enzyme replacement therapy and quality of life in adults with late-onset Pompe disease in China: A retrospective matched cohort study

Huanyu Zhang, Shanquan Chen, [ ... ], Dong Dong

Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II

Alex J. Shamoun, Gisienne Reis, [ ... ], Leonardo F. Ferreira

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