Enzyme replacement for infantile Pompe disease: the first step toward a cure.
Wagner
Kathryn R
KR
D002648:Child; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy*; D006801:Humans; D010166:Palliative Care / Q000379:methods*; D011788:Quality of Life; D013727:Terminal Care / Q000379:methods*; D016896:Treatment Outcome; D000520:alpha-Glucosidases / Q000627:therapeutic use*
C509951:GAA protein, human; D000520:alpha-Glucosidases