A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease

Published 2018 View Full Article

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Title
A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease
Authors
Keywords
-
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 123, Issue 4, Pages 488-494
Publisher
Elsevier BV
Online
2018-02-13
DOI
10.1016/j.ymgme.2018.02.006

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