The effects of intracisternal enzyme replacement versus sham treatment on central neuropathology in preclinical canine fucosidosis

Conclusions: Despite reduced severity of fucosidosis neuropathology with partial enzyme replacement, more complete and sustained biochemical correction is required to halt neuropathological processes in this large animal model of lysosomal storage disease.
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Source Type: research