DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY

摘要

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B. Co 4, 4, £1) Co in in 0:, cNJ in in -C, Cu r Co 0) c a CN4 U) C a _ 0 0 Cu _ _ C Cl) a - a Co -C a U) in Cu in a CI) C-) ('3 Co ~> Ct) C) ~ ~t$ C) '-9