Article

Cerliponase alfa in the treatment of patients with classic and atypical late infantile neuronal ceroid lipofuscinosis in Latin America

2022-11-28

Abstract excerpt

<title>Abstract</title> <p><underline>Introduction:</underline> Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), is a neurodegenerative autosomal recessive disease caused by <italic>TPP1 </italic>gene<italic> </italic>variants<italic>, </italic>with a spectrum of classic and atypical phenotypes. The aim of treatment is to slow functional decline as early as possible, improving quality of life and surv...

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Literature Corpus work: be85d243-fe03-5d6c-8749-4339a87e4bf6
DOI: 10.21203/rs.3.rs-2159184/v1

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