Cerebrospinal fluid neurofilament light chain levels in CLN2 disease patients treated with enzyme replacement therapy normalise after two years on treatment

Classic late infantile neuronal ceroid lipofuscinosis (CLN2 disease) is caused by a deficiency of tripeptidyl-peptidase-1. In 2017, the first CLN2 enzyme replacement therapy (ERT) cerliponase alfa (Brineura) was approved by the FDA and EMA. The CLN2 disease clinical rating scale (CLN2 CRS) was devel...

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Detalles Bibliográficos
Autores principales: Iwan, Katharina, Patel, Nina, Heslegrave, Amanda, Borisova, Mina, Lee, Laura, Bower, Rebecca, Mole, Sara E., Mills, Philippa B., Zetterberg, Henrik, Mills, Kevin, Gissen, Paul, Heywood, Wendy E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: F1000 Research Limited 2022
Materias:
Brief Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8777495/
https://www.ncbi.nlm.nih.gov/pubmed/35106137
http://dx.doi.org/10.12688/f1000research.54556.2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8777495/
https://www.ncbi.nlm.nih.gov/pubmed/35106137
http://dx.doi.org/10.12688/f1000research.54556.2

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