ArticleMolecular genetics and metabolism2012

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 et al.PubMed ↗Full text ↗Publisher ↗

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