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

Issue title: Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease: Turin, Italy, November 21–22, 2014

Article type: Research Article

Authors: van der Meijden, J.C. | Güngör, D. | Kruijshaar, M.E. | Muir, A.D.J. | Broekgaarden, H.A. | van der Ploeg, A.T.

Affiliations: Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands | International Pompe Association, Association for Glycogen Storage Disease UK, UK | International Pompe Association, Vereniging Spierziekten Nederland, Netherlands

Note: [] Correspondence to: J.C. van der Meijden, Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Dr. Molewaterplein 60, Room Sb-1629, 3000 CB Rotterdam, Netherlands. E-mail: c.vandermeijden.1@erasmusmc.nl

DOI: 10.3233/JND-159032

Journal: Journal of Neuromuscular Diseases, vol. 2, no. s1, pp. S35-S35, 2015