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45. The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders. Henley WE; Anderson LJ; Wyatt KM; Nikolaou V; Anderson R; Logan S J Inherit Metab Dis; 2014 Nov; 37(6):939-44. PubMed ID: 24519353 [TBL] [Abstract][Full Text] [Related]
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