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Ratko TA, Marbella A, Godfrey S, et al. Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2013 Jan. (Technical Briefs, No. 12.)

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Enzyme-Replacement Therapies for Lysosomal Storage Diseases [Internet].

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Appendix CSummaries of Published Registry Studies

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