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Time- and Dose-Dependent Normalization of Pathological Lysosomal Storage and Biochemistry in the Mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo Syndrome Type Β) Mouse Model by Intracerebroventricular Enzyme Replacement Therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 Fusion Pro

Molecular Genetics and Metabolism(2016)

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