Buffy Coat Score as a Biomarker of Treatment Response in Neuronal Ceroid Lipofuscinosis Type 2

The introduction of intracerebroventricular (ICV) enzyme replacement therapy (ERT) for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease has produced dramatic improvements in disease management. However, assessments of therapeutic effect for ICV ERT are limited to clinical observatio...

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Detalles Bibliográficos
Autores principales: Sivananthan, Siyamini, Lee, Laura, Anderson, Glenn, Csanyi, Barbara, Williams, Ruth, Gissen, Paul
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9954623/
https://www.ncbi.nlm.nih.gov/pubmed/36831752
http://dx.doi.org/10.3390/brainsci13020209

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9954623/
https://www.ncbi.nlm.nih.gov/pubmed/36831752
http://dx.doi.org/10.3390/brainsci13020209

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