Publication | Open Access

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

Preclinical Mouse ModelRecombinant Palmitoyl–protein ThioesteraseVisceral Lysosomal StorageMedicinePathologyPharmacologyCell BiologyLysosomal Storage DiseaseMolecular Medicine