Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten's disease, is a rare, autosomal recessive, pediatric neurodegenerative disease resulting from pathogenic variants in the gene encoding lysosomal enzyme tripeptidyl peptidase 1 (TPP1). A deficiency of TPP1 results in accumulation…

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