Development of the “Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease” Based on 6 Years Treatment Experience in 48 Patients

Intracerebroventricular enzyme replacement therapy (ICV-ERT) for CLN2 disease represents the first approved treatment for neuronal ceroid lipofuscinosis (NCL) diseases. It is the first treatment where a recombinant lysosomal enzyme, cerliponase alfa, is administered into the lateral cerebral ventric...

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Detalles Bibliográficos
Autores principales: Schwering, Christoph, Kammler, Gertrud, Wibbeler, Eva, Christner, Martin, Knobloch, Johannes K.-M., Nickel, Miriam, Denecke, Jonas, Baehr, Michael, Schulz, Angela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8255505/
https://www.ncbi.nlm.nih.gov/pubmed/33543660
http://dx.doi.org/10.1177/0883073821989154

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