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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 Birnbaum, Denis S Yilmaz, Barbara Streit, Ewelina M Lenartowicz, Thomas C.M. Thompson, Jonathan Cooper, Sandra Hofmann
2012-09-01
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