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Lancet 374:1986-1996, 1950, Mehta,A.,et al, 2009
Glycogen-Storage Disease Type II
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Insights Into the Diagnosis and Treatment of Lysosomal Storage Diseases
Arch Neurol 60:322-328, Wegner,D.A.,et al, 2003
Enzyme Replacement Therapy in Fabry Disease
JAMA 285:2743-2749, Schiffmann,R.,et al, 2001
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Long-Term Benefit of Enzyme Replacement Therapy in Pompe Disease
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Neurol 86:e190-e194, McIntosh, P. & Karam, C., 2016