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Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial

Publication ,  Conference
Schoser, B; Bratkovic, D; Byrne, BJ; Clemens, PR; Goker-Alpan, O; Kishnani, P; Ming, X; Roberts, M; Schwenkreis, P; Sivakumar, K; Wright, J ...
Published in: Molecular Genetics and Metabolism
February 2019

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2019

Volume

126

Issue

2

Start / End Page

S132 / S133

Publisher

Elsevier BV

Related Subject Headings

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

Citation

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Schoser, B., Bratkovic, D., Byrne, B. J., Clemens, P. R., Goker-Alpan, O., Kishnani, P., … van der Ploeg, A. T. (2019). Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial. In Molecular Genetics and Metabolism (Vol. 126, pp. S132–S133). Elsevier BV. https://doi.org/10.1016/j.ymgme.2018.12.340
Schoser, Benedikt, Drago Bratkovic, Barry J. Byrne, Paula R. Clemens, Ozlem Goker-Alpan, Priya Kishnani, Xue Ming, et al. “Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial.” In Molecular Genetics and Metabolism, 126:S132–33. Elsevier BV, 2019. https://doi.org/10.1016/j.ymgme.2018.12.340.
Schoser B, Bratkovic D, Byrne BJ, Clemens PR, Goker-Alpan O, Kishnani P, et al. Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial. In: Molecular Genetics and Metabolism. Elsevier BV; 2019. p. S132–3.
Schoser, Benedikt, et al. “Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial.” Molecular Genetics and Metabolism, vol. 126, no. 2, Elsevier BV, 2019, pp. S132–33. Crossref, doi:10.1016/j.ymgme.2018.12.340.
Schoser B, Bratkovic D, Byrne BJ, Clemens PR, Goker-Alpan O, Kishnani P, Ming X, Roberts M, Schwenkreis P, Sivakumar K, Wright J, Sitaraman S, Barth JA, Lagast H, Mozaffar T, van der Ploeg AT. Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial. Molecular Genetics and Metabolism. Elsevier BV; 2019. p. S132–S133.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2019

Volume

126

Issue

2

Start / End Page

S132 / S133

Publisher

Elsevier BV

Related Subject Headings

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