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Molecular Genetics and Metabolism|May 2, 2017
The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)P Harmatz, C J Hendriksz, C Lampe, et al.Bone Marrow Transplantation|June 11, 2003
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sourcesG Souillet, N Guffon, I Maire, et al.Molecular Genetics and Metabolism|May 10, 2011
Comprehensive cDNA study and quantitative analysis of mutant HADHA and HADHB transcripts in a French cohort of 52 patients with mitochondrial trifunctional protein deficiencyA Boutron, C Acquaviva, C Vianey-Saban, et al.Journal of Inherited Metabolic Disease|June 20, 2008
Diagnostic work-up and management of patients with isolated methylmalonic acidurias in European metabolic centresT Zwickler, M Lindner, H I Aydin, et al.Journal of Inherited Metabolic Disease|July 31, 2009
Prediction of outcome in isolated methylmalonic acidurias: combined use of clinical and biochemical parametersF Hörster, S F Garbade, T Zwickler, et al.Journal of Inherited Metabolic Disease|June 7, 2012
Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapyE Braunlin, H Rosenfeld, C Kampmann, et al.Journal of Inherited Metabolic Disease|March 16, 2013
Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose studyL Borgwardt, C I Dali, J Fogh, et al.Pageof 4