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  1. Article

    169 Update on Enzyme Replacement Therapy (ERT) with Recombinant Human Arylsulfatase B (RHASB) for MPS VI (Maroteaux-Lamy)

    MPS VI is a rare, life-threatening lysosomal storage disease with no effective treatment. ERT with rhASB has shown promising results in 2 clinical studies. The objective of this Phase 3 study was to confirm ef...

    P Harmatz, R Giugliani, I Schwartz, N Guffon, CSA Miranda, E Teles… in Pediatric Research (2005)

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