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NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD)

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; Lu, X ...
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

S34 / S34

Publisher

Elsevier BV

Related Subject Headings

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

Citation

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Dimachkie, M. M., Barohn, R. J., Byrne, B., Goker-Alpan, O., Kishnani, P. S., Ladha, S., … Benedikt Schoser; on behalf of NEO-EXT investigators, . (2021). NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD). In Molecular Genetics and Metabolism (Vol. 132, pp. S34–S34). Elsevier BV. https://doi.org/10.1016/j.ymgme.2020.12.064
Dimachkie, Mazen M., Richard J. Barohn, Barry Byrne, Ozlem Goker-Alpan, Priya S. Kishnani, Shafeeq Ladha, Pascal Laforêt, et al. “NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD).” In Molecular Genetics and Metabolism, 132:S34–S34. Elsevier BV, 2021. https://doi.org/10.1016/j.ymgme.2020.12.064.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, et al. NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD). In: Molecular Genetics and Metabolism. Elsevier BV; 2021. p. S34–S34.
Dimachkie, Mazen M., et al. “NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD).” Molecular Genetics and Metabolism, vol. 132, no. 2, Elsevier BV, 2021, pp. S34–S34. Crossref, doi:10.1016/j.ymgme.2020.12.064.
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, Ivanina I, Lu X, Benedikt Schoser; on behalf of NEO-EXT investigators. NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD). Molecular Genetics and Metabolism. Elsevier BV; 2021. p. S34–S34.
Journal cover image

Published In

Molecular Genetics and Metabolism

DOI

ISSN

1096-7192

Publication Date

February 2021

Volume

132

Issue

2

Start / End Page

S34 / S34

Publisher

Elsevier BV

Related Subject Headings

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