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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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Dettagli Bibliografici
Pubblicato in:Ther Clin Risk Manag
Autori principali: Specchio, Nicola, Pietrafusa, Nicola, Trivisano, Marina
Natura: Artigo
Lingua:Inglês
Pubblicazione: Dove 2020
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Accesso online: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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