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]
Producer: 20160325Description: 495-503 p. digitalISSN:- 1573-2665
- Adolescent
- Adult
- Aged
- Aged, 80 and over
- Child
- Child, Preschool
- Cohort Studies
- Enzyme Replacement Therapy -- methods
- Fatigue
- Female
- Glycogen Storage Disease Type II -- drug therapy
- Humans
- Infant
- Infant, Newborn
- Male
- Middle Aged
- Patient Outcome Assessment
- Patient Participation
- Quality of Life
- Self Report
- Young Adult
- alpha-Glucosidases -- therapeutic use
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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