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.

Autor: Meijden, J., Güngör, D., Kruijshaar, M., Muir, A., Broekgaarden, H., Ploeg, A.

Publikováno v: Journal of Inherited Metabolic Disease; May2015, Vol. 38 Issue 3, p495-503, 9p

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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.

Autor: van der Meijden JC; Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands., Güngör D; Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands., Kruijshaar ME; Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands., Muir AD; International Pompe Association, Association for Glycogen Storage Disease UK, UK., Broekgaarden HA; International Pompe Association, Vereniging Spierziekten Nederland, Netherlands., van der Ploeg AT; Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands.

Publikováno v: Journal of neuromuscular diseases [J Neuromuscul Dis] 2015; Vol. 2 (s1), pp. S35.