TY - JOUR AU - de Vries, Juna M. AU - van der Beek, Nadine AME AU - Hop, Wim CJ AU - Karstens, Francois PJ AU - Wokke, John H. AU - de Visser, Marianne AU - van Engelen, Baziel GM AU - Kuks, Jan BM AU - van der Kooi, Anneke J. AU - Notermans, Nicolette C. AU - Faber, Catharina G. AU - Verschuuren, Jan JGM AU - Kruijshaar, Michelle E. AU - Reuser, Arnold JJ AU - van Doorn, Pieter A. AU - van der Ploeg, Ans T. PY - 2012 DA - 2012/09/26 TI - Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study JO - Orphanet Journal of Rare Diseases SP - 73 VL - 7 IS - 1 AB - Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscular disorder, is of promising but variable efficacy. We investigated whether it alters the course of disease, and also identified potential prognostic factors. SN - 1750-1172 UR - https://doi.org/10.1186/1750-1172-7-73 DO - 10.1186/1750-1172-7-73 ID - de Vries2012 ER -