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Mini-COMET study: Safety, immunogenicity, and preliminary efficacy for repeat avalglucosidase alfa dosing in patients with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa and demonstrated clinical decline

Publication ,  Conference
Kronn, D; Brassier, A; Broomfield, A; Davison, J; Hahn, S; Kishnani, PS; Kumada, S; Labarthe, F; Ohki, H; Pichard, S; Haack, KA; Fleurinck, C ...
Published in: Molecular Genetics and Metabolism
February 2020

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S92 / S92

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences
 

Citation

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Kronn, D., Brassier, A., Broomfield, A., Davison, J., Hahn, S., Kishnani, P. S., … Johnson, J. (2020). Mini-COMET study: Safety, immunogenicity, and preliminary efficacy for repeat avalglucosidase alfa dosing in patients with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa and demonstrated clinical decline. In Molecular Genetics and Metabolism (Vol. 129, pp. S92–S92). Elsevier BV. https://doi.org/10.1016/j.ymgme.2019.11.231
Kronn, David, Anaïs Brassier, Alexander Broomfield, James Davison, Sihoun Hahn, Priya S. Kishnani, Satoko Kumada, et al. “Mini-COMET study: Safety, immunogenicity, and preliminary efficacy for repeat avalglucosidase alfa dosing in patients with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa and demonstrated clinical decline.” In Molecular Genetics and Metabolism, 129:S92–S92. Elsevier BV, 2020. https://doi.org/10.1016/j.ymgme.2019.11.231.
Kronn D, Brassier A, Broomfield A, Davison J, Hahn S, Kishnani PS, Kumada S, Labarthe F, Ohki H, Pichard S, Haack KA, Fleurinck C, He S, Johnson J. Mini-COMET study: Safety, immunogenicity, and preliminary efficacy for repeat avalglucosidase alfa dosing in patients with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa and demonstrated clinical decline. Molecular Genetics and Metabolism. Elsevier BV; 2020. p. S92–S92.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S92 / S92

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences