Presymptomatic treatment of classic late-infantile neuronal ceroid lipofuscinosis with cerliponase alfa

BACKGROUND: Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare rapidly progressive neurodegenerative disorder, resulting in early death. Intracerebroventricular enzyme replacement therapy (ERT) with cerliponase alfa is now available and has shown to delay disease progression in symptomat...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Schaefers, J., van der Giessen, L. J., Klees, C., Jacobs, E. H., Sieverdink, S., Dremmen, M. H. G., Spoor, J. K. H., van der Ploeg, A. T., van den Hout, J. M. P., Huidekoper, H. H.
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2021
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8120778/
https://ncbi.nlm.nih.gov/pubmed/33990214
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01858-6
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