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Results 1 - 1 of 1 for Neuronal ceroid lipofuscinosis 1
  1. ... able to crawl or walk in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease; an inherited disease of the nervous system), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Cerliponase alfa is in a class ...