000 01743 a2200517 4500
005 20250516225755.0
264 0 _c20160325
008 201603s 0 0 eng d
022 _a1573-2665
024 7 _a10.1007/s10545-014-9751-2
_2doi
040 _aNLM
_beng
_cNLM
100 1 _avan der Meijden, J C
245 0 0 _aTen years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease.
_h[electronic resource]
260 _bJournal of inherited metabolic disease
_cMay 2015
300 _a495-503 p.
_bdigital
500 _aPublication Type: Journal Article; Research Support, Non-U.S. Gov't
650 0 4 _aAdolescent
650 0 4 _aAdult
650 0 4 _aAged
650 0 4 _aAged, 80 and over
650 0 4 _aChild
650 0 4 _aChild, Preschool
650 0 4 _aCohort Studies
650 0 4 _aEnzyme Replacement Therapy
_xmethods
650 0 4 _aFatigue
650 0 4 _aFemale
650 0 4 _aGlycogen Storage Disease Type II
_xdrug therapy
650 0 4 _aHumans
650 0 4 _aInfant
650 0 4 _aInfant, Newborn
650 0 4 _aMale
650 0 4 _aMiddle Aged
650 0 4 _aPatient Outcome Assessment
650 0 4 _aPatient Participation
650 0 4 _aQuality of Life
650 0 4 _aSelf Report
650 0 4 _aYoung Adult
650 0 4 _aalpha-Glucosidases
_xtherapeutic use
700 1 _aGüngör, D
700 1 _aKruijshaar, M E
700 1 _aMuir, A D J
700 1 _aBroekgaarden, H A
700 1 _avan der Ploeg, A T
773 0 _tJournal of inherited metabolic disease
_gvol. 38
_gno. 3
_gp. 495-503
856 4 0 _uhttps://doi.org/10.1007/s10545-014-9751-2
_zAvailable from publisher's website
999 _c24088005
_d24088005