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

  title={Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human-Glucosidase From Milk},
  author={Johanna M. P. Van den Hout and Joep H. J. Kamphoven and L{\'e}on P. F. Winkel and Willem Frans M. Arts and Johannes B. C. De Klerk and M. Christa B. Loonen and Arnold Vulto and Adri H. Cromme-Dijkhuis and Nynke Weisglas-Kuperus and Wim C. J. Hop and Hans Van Hirtum and Otto P. van Diggelen and Marijke A Boer and Marian A. Kroos and Pieter A. van Doorn and Edwin Van der Voort and Barbara Sibbles and Emiel J. J. M. Van Corven and Just P. J. Brakenhoff and Johan L. K. Van Hove and Jan A M Smeitink and Gerard de Jong and Arnold J. J. Reuser and Ans T. van der Ploeg},
Objective. Recent reports warn that the worldwide cell culture capacity is insufficient to fulfill the increasing demand for human protein drugs. Production in milk of transgenic animals is an attractive alternative. Kilogram quantities of product per year can be obtained at relatively low costs, even in small animals such as rabbits. We tested the long-term safety and efficacy of recombinant human -glucosidase (rhAGLU) from rabbit milk for the treatment of the lysosomal storage disorder Pompe… CONTINUE READING


Publications citing this paper.
Showing 1-10 of 17 extracted citations


Publications referenced by this paper.
Showing 1-10 of 48 references

Morphological changes in muscle tissue of patients with infantile Pompe’s disease receiving enzyme replacement therapy

LPF Winkel, JHJ Kamphoven, JMP Van den Hout
Muscle Nerve • 2003

Preliminary findings in patients with juvenile Pompe’s disease treated with recombinant human alpha-glucosidase from rabbit milk

LPF Winkel, JMP Van den Hout, JHJ Kamphoven
Am J Hum Genet • 2001

Similar Papers

Loading similar papers…