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Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa.
Mol Genet Metab Rep. 2024 Feb 1;38:101060. doi: 10.1016/j.ymgmr.2024.101060. eCollection 2024 Mar.
Mol Genet Metab Rep. 2024.
PMID: 38469103
Free PMC article.
"Real world effectiveness of cerliponase alfa in classical and atypical patients. A case series".
Espitia Segura OM, Hernández Z, Mancilla NI, Naranjo RA, Tavera L.
Espitia Segura OM, et al. Among authors: mancilla ni.
Mol Genet Metab Rep. 2021 Feb 3;27:100718. doi: 10.1016/j.ymgmr.2021.100718. eCollection 2021 Jun.
Mol Genet Metab Rep. 2021.
PMID: 33604240
Free PMC article.
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