Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Published 2015 View Full Article

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Title
Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency
Authors
Keywords
Olipudase alfa, Recombinant human acid sphingomyelinase, Dose escalation, Nonneuronopathic ASMD, Niemann–Pick disease type B
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 116, Issue 1-2, Pages 88-97
Publisher
Elsevier BV
Online
2015-05-30
DOI
10.1016/j.ymgme.2015.05.013

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