Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Published 2015 View Full Article

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Title
Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
Authors
Keywords
Enzyme replacement therapy, Palmitoyl protein thioesterase, Lysosomal storage disorder, Batten disease, Infantile neuronal ceroid lipofuscinosis
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 116, Issue 1-2, Pages 98-105
Publisher
Elsevier BV
Online
2015-05-12
DOI
10.1016/j.ymgme.2015.05.005

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