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.

Authors

Si Houn Hahn
David Kronn
Nancy D Leslie
Loren D M Pena
Pranoot Tanpaiboon, George Washington University
Michael J Gambello
James B Gibson
Richard Hillman
David W Stockton
John W Day
Raymond Y Wang
Kristina An Haack
Raheel Shafi
Susan Sparks
Yang Zhao
Catherine Wilson
Priya S Kishnani

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

APA Citation

Hahn, S., Kronn, D., Leslie, N., Pena, L., Tanpaiboon, P., Gambello, M., Gibson, J., Hillman, R., Stockton, D., Day, J., Wang, R., An Haack, K., Shafi, R., Sparks, S., Zhao, Y., Wilson, C., & Kishnani, P. (2018). 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.. Genetics in medicine : official journal of the American College of Medical Genetics, (). http://dx.doi.org/10.1038/gim.2018.2

Peer Reviewed

1