Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease

Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is an ultra‐rare pediatric neurodegenerative disorder characterized by deficiency of the lysosomal enzyme tripeptidyl peptidase‐1 (TPP1). In the absence of adequate TPP1, lysosomal storage material accumulation occurs in the central nervous system...

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Detalles Bibliográficos
Autores principales: Hammon, Kevin, de Hart, Greg, Vuillemenot, Brian R., Kennedy, Derek, Musson, Don, O’Neill, Charles A., Katz, Martin L., Henshaw, Joshua W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8504808/
https://www.ncbi.nlm.nih.gov/pubmed/34076336
http://dx.doi.org/10.1111/cts.13028

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