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NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients

Publication ,  Conference
Dimachkie, MM; Barohn, RJ; Byrne, B; Goker-Alpan, O; Kishnani, PS; Ladha, S; Laforêt, P; Mengel, KE; Pena, LDM; Sacconi, S; Straub, V; Liu, K ...
Published in: Molecular Genetics and Metabolism
February 2020

Duke Scholars

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Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S49 / S49

Publisher

Elsevier BV

Related Subject Headings

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

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Dimachkie, M. M., Barohn, R. J., Byrne, B., Goker-Alpan, O., Kishnani, P. S., Ladha, S., … Liu, K. (2020). NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients. In Molecular Genetics and Metabolism (Vol. 129, pp. S49–S49). Elsevier BV. https://doi.org/10.1016/j.ymgme.2019.11.107
Dimachkie, Mazen M., Richard J. Barohn, Barry Byrne, Ozlem Goker-Alpan, Priya S. Kishnani, Shafeeq Ladha, Pascal Laforêt, et al. “NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients.” In Molecular Genetics and Metabolism, 129:S49–S49. Elsevier BV, 2020. https://doi.org/10.1016/j.ymgme.2019.11.107.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, et al. NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients. In: Molecular Genetics and Metabolism. Elsevier BV; 2020. p. S49–S49.
Dimachkie, Mazen M., et al. “NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients.” Molecular Genetics and Metabolism, vol. 129, no. 2, Elsevier BV, 2020, pp. S49–S49. Crossref, doi:10.1016/j.ymgme.2019.11.107.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Pena LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg A, Vissing J, Young P, Haack KA, Fleurinck C, Johnson J, Liu K. NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients. Molecular Genetics and Metabolism. Elsevier BV; 2020. p. S49–S49.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2020

Volume

129

Issue

2

Start / End Page

S49 / S49

Publisher

Elsevier BV

Related Subject Headings

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