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

Author(s):
Vries, J.M. de; Beek, N.A.M.E. van der; Hop, W.C.J.; Karstens, F.P.J.; Wokke, J.H.; Visser, M. de; Engelen, B.G.M. van; Kuks, J.B.M.; Kooi, A.J. van der; Notermans, N.C.; Faber, C.G.; Verschuuren, J.J.G.M. (0000-0002-4572-1501); Kruijshaar, M.E.; Reuser, A.J.J.; Doorn, P.A. van; Ploeg, A.T. van der
Published
2012
Publication type
Article / Letter to editor
Pompe diseaseGlycogen storage disease type IIOMIM number 232300Acid alpha-glucosidaseAlglucosidase alfaEnzyme replacement therapyLysosomal storage disorderMuscle strengthLung function
Date issued
2012-09-26
Language
English
Is part of
Orphanet Journal of Rare Diseases (2012), Vol. 7, p. -
hdl
1887/97114
doi
10.1186/1750-1172-7-73