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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8120778/
https://www.ncbi.nlm.nih.gov/pubmed/33990214
http://dx.doi.org/10.1186/s13023-021-01858-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8120778/
https://www.ncbi.nlm.nih.gov/pubmed/33990214
http://dx.doi.org/10.1186/s13023-021-01858-6

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