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Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline

Publication ,  Conference
Kronn, D; Davison, J; Brassier, A; Broomfield, A; Hahn, SH; Kumada, S; Labarthe, F; Ohki, H; Prakalapakorn, SG; Haack, KA; Meng, X; Sparks, S ...
Published in: Molecular Genetics and Metabolism
February 2022

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2022

Volume

135

Issue

2

Start / End Page

S68 / S68

Publisher

Elsevier BV

Related Subject Headings

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

Citation

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Kronn, D., Davison, J., Brassier, A., Broomfield, A., Hahn, S. H., Kumada, S., … Kishnani, P. S. (2022). Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline. In Molecular Genetics and Metabolism (Vol. 135, pp. S68–S68). Elsevier BV. https://doi.org/10.1016/j.ymgme.2021.11.171
Kronn, David, James Davison, Anaïs Brassier, Alexander Broomfield, Si Houn Hahn, Satoko Kumada, François Labarthe, et al. “Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline.” In Molecular Genetics and Metabolism, 135:S68–S68. Elsevier BV, 2022. https://doi.org/10.1016/j.ymgme.2021.11.171.
Kronn D, Davison J, Brassier A, Broomfield A, Hahn SH, Kumada S, Labarthe F, Ohki H, Prakalapakorn SG, Haack KA, Meng X, Sparks S, Tammireddy S, Wilson C, Zaher A, Zhou T, Chien Y-H, Kishnani PS. Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline. Molecular Genetics and Metabolism. Elsevier BV; 2022. p. S68–S68.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2022

Volume

135

Issue

2

Start / End Page

S68 / S68

Publisher

Elsevier BV

Related Subject Headings

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