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Author Title [ Type(Asc)] Year
Filters: Author is Guffon, N.  [Clear All Filters]
Journal Article
Swiedler, S. J., Beck, M., Bajbouj, M., Giugliani, R., et al. (2005). Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). American Journal of Medical Genetics, 134 A(2), 144 - 150.
Karageorgos, L., Brooks, D. A., Pollard, A., Melville, E. L., et al. (2007). Mutational analysis of 105 mucopolysaccharidosis type VI patients. Human Mutation, 28(9), 897 - 903.
Harmatz, P., Giugliani, R., D. Schwartz, I. V., Guffon, N., et al. (2008). Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Molecular Genetics and Metabolism, 94(4), 469 - 475.
Braunlin, E., Rosenfeld, H., Kampmann, C., Johnson, J., et al. (2013). Enzyme replacement therapy for mucopolysaccharidosis VI: Long-term cardiac effects of galsulfase (Naglazyme®) therapy. Journal of Inherited Metabolic Disease, 36(2), 385 - 394.
Decker, C., Yu, Z. - F., Giugliani, R., Schwartz, I. V. D., et al. (2010). Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. Journal of Pediatric Rehabilitation Medicine, 3(2), 89 - 100.
Harmatz, P., Yu, Z. - F., Giugliani, R., Schwartz, I. V. D., et al. (2010). Enzyme replacement therapy for mucopolysaccharidosis VI: Evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. Journal of Inherited Metabolic Disease, 33(1), 51 - 60.
Harmatz, P., Giugliani, R., Schwartz, I., Guffon, N., et al. (2006). Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-l. Journal of Pediatrics, 148(4), 533 - 533.e.
Harmatz, P., Ketteridge, D., Giugliani, R., Guffon, N., et al. (2005). Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): Results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylga. Pediatrics, 115(6), e681 - e689.

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