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Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses
The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal storage disorders caused by mutations in at least 13 different genes and primarily affect the brain and the retina of children or young adults. The disorders are characterized by progressive neurological deterioration...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer International Publishing
2019
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6440934/ https://www.ncbi.nlm.nih.gov/pubmed/30877620 http://dx.doi.org/10.1007/s40263-019-00620-8 |