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Number of items: 4.

Braunlin, E; Rosenfeld, H; Kampmann, C; Johnson, J; Beck, M; Giugliani, R; Guffon, N; Ketteridge, D; Sá Miranda, C M; Scarpa, M; Schwartz, I V; Leão Teles, E; Wraith, J E; Barrios, P; Dias da Silva, E; Kurio, G; Richardson, M; Gildengorin, G; Hopwood, J J; Imperiale, M; Schatz, A; Decker, C; Harmatz, P (2013). Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy. Journal of Inherited Metabolic Disease, 36(2):385-394.

Wraith, J E; Guffon, N; Rohrbach, M; Hwu, W L; Korenke, G C; Bembi, B; Luzy, C; Giorgino, R; Sedel, F (2009). Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study. Molecular Genetics and Metabolism, 98(3):250-254.

Hörster, F; Garbade, S F; Zwickler, T; Aydin, H I; Bodamer, O A; Burlina, A B; Das, A M; De Klerk, J B; Dionisi-Vici, C; Geb, S; Gökcay, G; Guffon, N; Maier, E M; Morava, E; Walter, J H; Schwan, B; Wijburg, F A; Lindner, M; Grünewald, S; Baumgartner, M R; Kölker, S (2009). Prediction of outcome in isolated methylmalonic acidurias: combined use of clinical and biochemical parameters. Journal of Inherited Metabolic Disease, 32(5):630-639.

Zwickler, T; Lindner, M; Aydin, H I; Baumgartner, M R; Bodamer, O A; Burlina, A B; Das, A M; DeKlerk, J B C; Gökcay, G; Grünewald, S; Guffon, N; Maier, E M; Morava, E; Geb, S; Schwahn, B; Walter, J H; Wendel, U; Wijburg, F A; Müller, E; Kölker, S; Hörster, F (2008). Diagnostic work-up and management of patients with isolated methylmalonic acidurias in European metabolic centres. Journal of Inherited Metabolic Disease, 31(3):361-367.

This list was generated on Tue Sep 26 19:20:54 2017 CEST.