Title

Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.

Document Type

Journal Article

Publication Date

3-22-2018

Journal

Genetics in medicine : official journal of the American College of Medical Genetics

DOI

10.1038/gim.2018.2

Peer Reviewed

1

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