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Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry Disease

Elsewhere, we reported the safety and efficacy results of a multicenter phase 3 trial of recombinant human α-galactosidase A (rh-αGalA) replacement in patients with Fabry disease. All 58 patients who were enrolled in the 20-wk phase 3 double-blind, randomized, and placebo-controlled study received s...

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Autors principals: Wilcox, William R., Banikazemi, Maryam, Guffon, Nathalie, Waldek, Stephen, Lee, Philip, Linthorst, Gabor E., Desnick, Robert J., Germain, Dominique P.
Format: Artigo
Idioma:Inglês
Publicat: The American Society of Human Genetics 2004
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC1182009/
https://ncbi.nlm.nih.gov/pubmed/15154115
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