Article
Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).
Journal of neuroscience research - 1 Nov 2014
Katz Martin L, Coates Joan R, Sibigtroth Christine M, Taylor Jacob D, Carpentier Melissa, Young Whitney M, Wininger Fred A, Kennedy Derek, Vuillemenot Brian R, O'Neill Charles A
Abstract excerpt
Using a canine model of classical late-infantile neuronal ceroid lipofuscinosis (CLN2 disease), a study was conducted to evaluate the potential pharmacological activity of recombinant human tripeptidyl peptidase-1 (rhTPP1) enzyme replacement therapy administered directly to the cerebrospinal flui...
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