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Therapeutic landscape for Batten disease: current treatments and future prospects

Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of devastating lysosomal storage disorders that collectively represent the most common inherited paediatric neurodegenerative disorders worldwide. Batten disease can result from mutations in 1 of 13 genes. These mutat...

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Detalhes bibliográficos
Publicado no:Nat Rev Neurol
Main Authors: Johnson, Tyler B., Cain, Jacob T., White, Katherine A., Ramirez-Montealegre, Denia, Pearce, David A., Weimer, Jill M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6681450/
https://ncbi.nlm.nih.gov/pubmed/30783219
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41582-019-0138-8
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