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Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy
Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a progressive neurodegenerative disease that results in early-onset, severe, progressive, neurological disabilities, leading to death in late childhood or early adolescence. Management has relied on symptomatic care, and supportive and palliati...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Dove
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7127909/ https://www.ncbi.nlm.nih.gov/pubmed/32280231 http://dx.doi.org/10.2147/TCRM.S241048 |