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publicationDate 2016-05-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2016120957-A1
titleOfInvention Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
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.
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