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Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial

Publication ,  Conference
Clemens, PR; Mozaffar, T; Schoser, B; Bratkovic, D; Byrne, BJ; Goker-Alpan, O; Roberts, M; Schwenkreis, P; Sivakumar, K; van der Ploeg, AT ...
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

S40 / S41

Publisher

Elsevier BV

Related Subject Headings

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

Citation

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Clemens, P. R., Mozaffar, T., Schoser, B., Bratkovic, D., Byrne, B. J., Goker-Alpan, O., … Ming, X. (2019). Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial. In Molecular Genetics and Metabolism (Vol. 126, pp. S40–S41). Elsevier BV. https://doi.org/10.1016/j.ymgme.2018.12.084
Clemens, Paula R., Tahseen Mozaffar, Benedikt Schoser, Drago Bratkovic, Barry J. Byrne, Ozlem Goker-Alpan, Mark Roberts, et al. “Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial.” In Molecular Genetics and Metabolism, 126:S40–41. Elsevier BV, 2019. https://doi.org/10.1016/j.ymgme.2018.12.084.
Clemens PR, Mozaffar T, Schoser B, Bratkovic D, Byrne BJ, Goker-Alpan O, et al. Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial. In: Molecular Genetics and Metabolism. Elsevier BV; 2019. p. S40–1.
Clemens, Paula R., et al. “Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial.” Molecular Genetics and Metabolism, vol. 126, no. 2, Elsevier BV, 2019, pp. S40–41. Crossref, doi:10.1016/j.ymgme.2018.12.084.
Clemens PR, Mozaffar T, Schoser B, Bratkovic D, Byrne BJ, Goker-Alpan O, Roberts M, Schwenkreis P, Sivakumar K, van der Ploeg AT, Wright J, Sitaraman S, Barth JA, Lagast H, Kishnani P, Ming X. Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial. Molecular Genetics and Metabolism. Elsevier BV; 2019. p. S40–S41.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2019

Volume

126

Issue

2

Start / End Page

S40 / S41

Publisher

Elsevier BV

Related Subject Headings

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