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Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction

Publication ,  Conference
Desai, AK; Li, C; Gupta, P; Dempsey, K; Bhambhani, V; Hopkin, R; Ficicioglu, C; Tanpaiboon, P; Craigen, WJ; KIshnani, PS
Published in: Molecular Genetics and Metabolism
February 2021

Duke Scholars

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2021

Volume

132

Issue

2

Start / End Page

S33 / S33

Publisher

Elsevier BV

Related Subject Headings

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

Citation

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Desai, A. K., Li, C., Gupta, P., Dempsey, K., Bhambhani, V., Hopkin, R., … KIshnani, P. S. (2021). Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction. In Molecular Genetics and Metabolism (Vol. 132, pp. S33–S33). Elsevier BV. https://doi.org/10.1016/j.ymgme.2020.12.062
Desai, Ankit K., Cindy Li, Punita Gupta, Katherine Dempsey, Vikas Bhambhani, Robert Hopkin, Can Ficicioglu, Pranoot Tanpaiboon, William J. Craigen, and Priya S. KIshnani. “Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction.” In Molecular Genetics and Metabolism, 132:S33–S33. Elsevier BV, 2021. https://doi.org/10.1016/j.ymgme.2020.12.062.
Desai AK, Li C, Gupta P, Dempsey K, Bhambhani V, Hopkin R, et al. Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction. In: Molecular Genetics and Metabolism. Elsevier BV; 2021. p. S33–S33.
Desai, Ankit K., et al. “Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction.” Molecular Genetics and Metabolism, vol. 132, no. 2, Elsevier BV, 2021, pp. S33–S33. Crossref, doi:10.1016/j.ymgme.2020.12.062.
Desai AK, Li C, Gupta P, Dempsey K, Bhambhani V, Hopkin R, Ficicioglu C, Tanpaiboon P, Craigen WJ, KIshnani PS. Transforming the clinical outcomes in CRIM-negative infantile Pompe disease identified via newborn screening: The benefits of early treatment with enzyme replacement therapy and immune tolerance induction. Molecular Genetics and Metabolism. Elsevier BV; 2021. p. S33–S33.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2021

Volume

132

Issue

2

Start / End Page

S33 / S33

Publisher

Elsevier BV

Related Subject Headings

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