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J Child Neurol. 2013 Sep;28(9):1159-68. doi: 10.1177/0883073813494480.

Treatment of the Ppt1(-/-) mouse model of infantile neuronal ceroid lipofuscinosis with the N-methyl-D-aspartate (NMDA) receptor antagonist memantine.

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Sanford Children's Health Research Center, Sanford Research/USD, Sioux Falls, SD, USA.


The neuronal ceroid lipofuscinoses, a family of neurodegenerative lysosomal storage disorders, represent the most common cause of pediatric-onset neurodegeneration. The infantile form has a devastatingly early onset and one of the fastest-progressing disease courses. Despite decades of research, the molecular mechanisms driving neuronal loss in infantile neuronal ceroid lipofuscinosis remain unknown. We have previously shown that N-methyl-d-aspartate (NMDA)-type glutamate receptors in the Ppt1(-/-) mouse model of this disease exhibit a hyperfunctional phenotype and postulate that aberrant glutamatergic activity may contribute to neural pathology in both the mouse model and human patients. To test this hypothesis, we treated Ppt1(-/-) mice with the NMDA receptor antagonist memantine and assessed their response to the drug using an accelerating rotarod. At 20 mg/kg, memantine treatment induced a delayed but notable improvement in Ppt1(-/-) mice. Much remains to be assessed before moving to patient trials, but these results suggest memantine has potential as a treatment.


Batten disease; NMDA receptor; infantile neuronal ceroid lipofuscinosis; memantine; rotarod

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