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
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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
Citation
APA
Chicago
ICMJE
MLA
NLM
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.
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