Výsledky vyhledávání - "H. A. Broekgaarden"

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: A.T. van der Ploeg, Deniz Güngör, Michelle E. Kruijshaar, J. C. van der Meijden, A. D. J. Muir, H. A. Broekgaarden

Publikováno v: Journal of Inherited Metabolic Disease, 38(3), 495-503. Springer Netherlands

Pompe disease is a rare, progressive lysosomal storage disorder for which enzyme therapy (ERT) became available in 2006. Four years earlier, the IPA/Erasmus MC survey, an international longitudinal prospective survey, was established to collect infor

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::245f001673ea20351a7c435ae45ab0c6
https://doi.org/10.1007/s10545-014-9751-2

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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: H. A. Broekgaarden, Deniz Güngör, A. D. J. Muir, A.T. van der Ploeg, J. C. van der Meijden, Michelle E. Kruijshaar

Publikováno v: Journal of Neuromuscular Diseases. 2:S35-S35

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::41c33a7e9797f04ae855bcc748de1b68
https://doi.org/10.3233/jnd-159032

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