van der Meijden, J C <br/><br/>
Ten 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.  [electronic resource] 

 -  Journal of inherited metabolic disease  May 2015 
 - 495-503 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1573-2665 
<br/><br/><label>Standard No.: </label>10.1007/s10545-014-9751-2  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Adolescent<br/>Adult<br/>Aged<br/>Aged, 80 and over<br/>Child<br/>Child, Preschool<br/>Cohort Studies<br/>Enzyme Replacement Therapy--methods<br/>Fatigue<br/>Female<br/>Glycogen Storage Disease Type II--drug therapy<br/>Humans<br/>Infant<br/>Infant, Newborn<br/>Male<br/>Middle Aged<br/>Patient Outcome Assessment<br/>Patient Participation<br/>Quality of Life<br/>Self Report<br/>Young Adult<br/>alpha-Glucosidases--therapeutic use