Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy

Published 2020 View Full Article

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Title
Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
Authors
Keywords
Acid sphingomyelin deficiency, Niemann-Pick Types A, B, /B, Lysosomal storage disorder, Disease burden
Journal
MOLECULAR GENETICS AND METABOLISM
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-06-24
DOI
10.1016/j.ymgme.2020.06.008

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