Associated Papers
Enzyme replacement therapy and fatigue in adults with Pompe disease.
Güngör D et al.
Molecular Genetics and Metabolism, 2013
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study.
Güngör D et al.
Orphanet Journal of Rare Diseases, 2013
Severely impaired health status at diagnosis of Pompe disease: a cross-sectional analysis to explore the potential utility of neonatal screening.
Rigter T et al.
Molecular Genetics and Metabolism, 2012
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