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Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT

Publication ,  Conference
Koeberl, D; Case, L; Smith, EC; Li, Y; Walters, C; Hornik, C; Thurberg, B; Bali, D; Bursac, N; Kishnani, PS
Published in: Molecular Genetics and Metabolism
February 2018

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2018

Volume

123

Issue

2

Start / End Page

S79 / S80

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 1103 Clinical Sciences
 

Citation

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Koeberl, D., Case, L., Smith, E. C., Li, Y., Walters, C., Hornik, C., … Kishnani, P. S. (2018). Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT. In Molecular Genetics and Metabolism (Vol. 123, pp. S79–S80). Elsevier BV. https://doi.org/10.1016/j.ymgme.2017.12.202
Koeberl, Dwight, Laura Case, Edward C. Smith, Yanzhen Li, Crista Walters, Christoph Hornik, Beth Thurberg, Deeksha Bali, Nenad Bursac, and Priya S. Kishnani. “Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT.” In Molecular Genetics and Metabolism, 123:S79–80. Elsevier BV, 2018. https://doi.org/10.1016/j.ymgme.2017.12.202.
Koeberl D, Case L, Smith EC, Li Y, Walters C, Hornik C, et al. Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT. In: Molecular Genetics and Metabolism. Elsevier BV; 2018. p. S79–80.
Koeberl, Dwight, et al. “Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT.” Molecular Genetics and Metabolism, vol. 123, no. 2, Elsevier BV, 2018, pp. S79–80. Crossref, doi:10.1016/j.ymgme.2017.12.202.
Koeberl D, Case L, Smith EC, Li Y, Walters C, Hornik C, Thurberg B, Bali D, Bursac N, Kishnani PS. Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT. Molecular Genetics and Metabolism. Elsevier BV; 2018. p. S79–S80.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2018

Volume

123

Issue

2

Start / End Page

S79 / S80

Publisher

Elsevier BV

Related Subject Headings

  • Genetics & Heredity
  • 1103 Clinical Sciences