Advances in the Treatment of Neuronal Ceroid Lipofuscinosis

Neuronal ceroid lipofuscinoses (NCL) represent a class of neurodegenerative disorders involving defective lysosomal processing enzymes or receptors, leading to lysosomal storage disorders, typically characterized by observation of cognitive and visual impairments, epileptic seizures, ataxia, and det...

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Bibliographic Details
Published in:Expert Opin Orphan Drugs
Main Authors: Rosenberg, Jonathan B., Chen, Alvin, Kaminsky, Stephen M., Crystal, Ronald G., Sondhi, Dolan
Format: Artigo
Language:Inglês
Published: 2019
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7755158/
https://ncbi.nlm.nih.gov/pubmed/33365208
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2019.1684258
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