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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...
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| Publié dans: | Ther Clin Risk Manag |
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| Auteurs principaux: | , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
Dove
2020
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7127909/ https://ncbi.nlm.nih.gov/pubmed/32280231 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TCRM.S241048 |
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