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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