Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

Barry J. Byrne, Benedikt Schoser, Priya S. Kishnani, Drago Bratkovic, Paula R. Clemens, Ozlem Goker-Alpan, Xue Ming, Mark Roberts, Matthias Vorgerd, Kumaraswamy Sivakumar, Ans T. van der Ploeg, Mitchell Goldman, Jacquelyn Wright, Fred Holdbrook, Vipul Jain, Elfrida R. Benjamin, Franklin Johnson, Sheela Sitaraman Das, Yasmine Wasfi, Tahseen Mozaffar

Open source

DOI: 10.1007/s00415-023-12096-0
Published: 2023-12-06
Container: Journal of Neurology
Publisher: Springer Science and Business Media LLC
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1007/s00415-023-12096-0,
  title = {Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)},
  author = {Barry J. Byrne and Benedikt Schoser and Priya S. Kishnani and Drago Bratkovic and Paula R. Clemens and Ozlem Goker-Alpan and Xue Ming and Mark Roberts and Matthias Vorgerd and Kumaraswamy Sivakumar and Ans T. van der Ploeg and Mitchell Goldman and Jacquelyn Wright and Fred Holdbrook and Vipul Jain and Elfrida R. Benjamin and Franklin Johnson and Sheela Sitaraman Das and Yasmine Wasfi and Tahseen Mozaffar},
  year = {2023},
  journal = {Journal of Neurology},
  doi = {10.1007/s00415-023-12096-0},
  url = {https://doi.org/10.1007/s00415-023-12096-0}
}

RIS

TY  - JOUR
TI  - Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)
AU  - Barry J. Byrne
AU  - Benedikt Schoser
AU  - Priya S. Kishnani
AU  - Drago Bratkovic
AU  - Paula R. Clemens
AU  - Ozlem Goker-Alpan
AU  - Xue Ming
AU  - Mark Roberts
AU  - Matthias Vorgerd
AU  - Kumaraswamy Sivakumar
AU  - Ans T. van der Ploeg
AU  - Mitchell Goldman
AU  - Jacquelyn Wright
AU  - Fred Holdbrook
AU  - Vipul Jain
AU  - Elfrida R. Benjamin
AU  - Franklin Johnson
AU  - Sheela Sitaraman Das
AU  - Yasmine Wasfi
AU  - Tahseen Mozaffar
PY  - 2023
JO  - Journal of Neurology
DO  - 10.1007/s00415-023-12096-0
UR  - https://doi.org/10.1007/s00415-023-12096-0
ER  -

APA

Byrne, B. J., Schoser, B., Kishnani, P. S., Bratkovic, D., Clemens, P. R., Goker-Alpan, O., Ming, X., Roberts, M., Vorgerd, M., Sivakumar, K., Ploeg, A. T. V. D., Goldman, M., Wright, J., Holdbrook, F., Jain, V., Benjamin, E. R., Johnson, F., Das, S. S., Wasfi, Y., & Mozaffar, T. (2023). Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02). Journal of Neurology. https://doi.org/10.1007/s00415-023-12096-0

Source records

crossref · retrieved 2026-09-26T06:26:58.939Z