Concepedia

Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease

Nadine A. M. E. van der Beek, M.L.C. Hagemans, Arnold Reuser, Wim C.J. Hop, Ans T. van der Ploeg, Pieter A. van Doorn, J.H.J. Wokke

Neuromuscular Disorders · 2008 · 109 citations · 20 references

DOI

Concepts

References

20

Recombinant human acid α-glucosidase

Priya S. Kishnani, Deya Corzo, Marc Nicolino et al. · Neurology · 2006 · 772 citations · Full text

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A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

Priya S. Kishnani, Wuh‐Liang Hwu, Hanna Mandel et al. · The Journal of Pediatrics · 2006 · 585 citations

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Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human α-Glucosidase From Milk

Johanna M. P. van den Hout, Joep H. J. Kamphoven, Léon P. F. Winkel et al. · PEDIATRICS · 2004 · 354 citations

, , +12

Recombinant human acid α-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

Andrea Amalfitano, A. Resai Bengur, Richard P. Morse et al. · Genetics in Medicine · 2001 · 347 citations · Full text

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Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling

M.G.E.M. Ausems, J. R. Verbiest, MMP Hermans et al. · European Journal of Human Genetics · 1999 · 304 citations

, , +13