Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Jie Hu, Jui-Yun Lu, Andrew M. S. Wong, Linda S. Hynan, Shari G. Birnbaum, Denis S. Yilmaz, Barbara M. Streit, Ewelina Lenartowicz, Thomas C. M. Thompson, Jonathan D. Cooper, Sandra L. Hofmann

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Medicine and Dentistry

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