Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

Juna M. de Vries; Nadine Anna Maria Elisabeth van der Beek; Wim C. J. Hop; Francois P. J. Karstens; John H. Wokke; Marianne de Visser; Baziel G. M. van Engelen; Jan B. M. Kuks; Anneke J. van der Kooi; Nicolette C. Notermans; Catharina G. Faber; Jan J. G. M. Verschuuren; Michelle E. Kruijshaar; Arnold J. J. Reuser; Pieter A. van Doorn; Ans T. van der Ploeg

doi:10.1186/1750-1172-7-73

Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

Juna M. de Vries, Nadine Anna Maria Elisabeth van der Beek, Wim C. J. Hop, Francois P. J. Karstens, John H. Wokke, Marianne de Visser, Baziel G. M. van Engelen, Jan B. M. Kuks, Anneke J. van der Kooi, Nicolette C. Notermans, Catharina G. Faber, Jan J. G. M. Verschuuren, Michelle E. Kruijshaar, Arnold J. J. Reuser, Pieter A. van Doorn, Ans T. van der Ploeg^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

Original language	English
Pages (from-to)	73
Journal	Orphanet Journal of Rare Diseases
Volume	7
DOIs	https://doi.org/10.1186/1750-1172-7-73
Publication status	Published - 26 Sep 2012

Keywords

Pompe disease
Glycogen storage disease type II
OMIM number 232300
Acid alpha-glucosidase
Alglucosidase alfa
Enzyme replacement therapy
Lysosomal storage disorder
Muscle strength
Lung function

Access to Document

10.1186/1750-1172-7-73

Cite this

de Vries, J. M., van der Beek, N. A. M. E., Hop, W. C. J., Karstens, F. P. J., Wokke, J. H., de Visser, M., van Engelen, B. G. M., Kuks, J. B. M., van der Kooi, A. J., Notermans, N. C., Faber, C. G., Verschuuren, J. J. G. M., Kruijshaar, M. E., Reuser, A. J. J., van Doorn, P. A., & van der Ploeg, A. T. (2012). Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study. Orphanet Journal of Rare Diseases, 7, 73. https://doi.org/10.1186/1750-1172-7-73

de Vries, Juna M. ; van der Beek, Nadine Anna Maria Elisabeth ; Hop, Wim C. J. ; Karstens, Francois P. J. ; Wokke, John H. ; de Visser, Marianne ; van Engelen, Baziel G. M. ; Kuks, Jan B. M. ; van der Kooi, Anneke J. ; Notermans, Nicolette C. ; Faber, Catharina G. ; Verschuuren, Jan J. G. M. ; Kruijshaar, Michelle E. ; Reuser, Arnold J. J. ; van Doorn, Pieter A. ; van der Ploeg, Ans T. / Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study. In: Orphanet Journal of Rare Diseases. 2012 ; Vol. 7. pp. 73.

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title = "Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study",

keywords = "Pompe disease, Glycogen storage disease type II, OMIM number 232300, Acid alpha-glucosidase, Alglucosidase alfa, Enzyme replacement therapy, Lysosomal storage disorder, Muscle strength, Lung function",

author = "{de Vries}, {Juna M.} and {van der Beek}, {Nadine Anna Maria Elisabeth} and Hop, {Wim C. J.} and Karstens, {Francois P. J.} and Wokke, {John H.} and {de Visser}, Marianne and {van Engelen}, {Baziel G. M.} and Kuks, {Jan B. M.} and {van der Kooi}, {Anneke J.} and Notermans, {Nicolette C.} and Faber, {Catharina G.} and Verschuuren, {Jan J. G. M.} and Kruijshaar, {Michelle E.} and Reuser, {Arnold J. J.} and {van Doorn}, {Pieter A.} and {van der Ploeg}, {Ans T.}",

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month = sep,

day = "26",

doi = "10.1186/1750-1172-7-73",

language = "English",

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journal = "Orphanet Journal of Rare Diseases",

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de Vries, JM, van der Beek, NAME, Hop, WCJ, Karstens, FPJ, Wokke, JH, de Visser, M, van Engelen, BGM, Kuks, JBM, van der Kooi, AJ, Notermans, NC, Faber, CG, Verschuuren, JJGM, Kruijshaar, ME, Reuser, AJJ, van Doorn, PA & van der Ploeg, AT 2012, 'Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study', Orphanet Journal of Rare Diseases, vol. 7, pp. 73. https://doi.org/10.1186/1750-1172-7-73

Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study. / de Vries, Juna M.; van der Beek, Nadine Anna Maria Elisabeth; Hop, Wim C. J.; Karstens, Francois P. J.; Wokke, John H.; de Visser, Marianne; van Engelen, Baziel G. M.; Kuks, Jan B. M.; van der Kooi, Anneke J.; Notermans, Nicolette C.; Faber, Catharina G.; Verschuuren, Jan J. G. M.; Kruijshaar, Michelle E.; Reuser, Arnold J. J.; van Doorn, Pieter A.; van der Ploeg, Ans T.

In: Orphanet Journal of Rare Diseases, Vol. 7, 26.09.2012, p. 73.

Research output: Contribution to journal › Article › Academic › peer-review

TY - JOUR

T1 - Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

AU - de Vries, Juna M.

AU - van der Beek, Nadine Anna Maria Elisabeth

AU - Hop, Wim C. J.

AU - Karstens, Francois P. J.

AU - Wokke, John H.

AU - de Visser, Marianne

AU - van Engelen, Baziel G. M.

AU - Kuks, Jan B. M.

AU - van der Kooi, Anneke J.

AU - Notermans, Nicolette C.

AU - Faber, Catharina G.

AU - Verschuuren, Jan J. G. M.

AU - Kruijshaar, Michelle E.

AU - Reuser, Arnold J. J.

AU - van Doorn, Pieter A.

AU - van der Ploeg, Ans T.

PY - 2012/9/26

Y1 - 2012/9/26

KW - Pompe disease

KW - Glycogen storage disease type II

KW - OMIM number 232300

KW - Acid alpha-glucosidase

KW - Alglucosidase alfa

KW - Enzyme replacement therapy

KW - Lysosomal storage disorder

KW - Muscle strength

KW - Lung function

U2 - 10.1186/1750-1172-7-73

DO - 10.1186/1750-1172-7-73

M3 - Article

VL - 7

SP - 73

JO - Orphanet Journal of Rare Diseases

JF - Orphanet Journal of Rare Diseases

SN - 1750-1172

ER -