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Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders

Neuronal Ceroid Lipofuscinosis (NCL), also known as Batten disease, is a group of genetically distinct lysosomal disorders that mainly affect the central nervous system, resulting in progressive motor and cognitive decline primarily in children. Multiple distinct genes involved in the metabolism of...

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Detalles Bibliográficos
Autores principales:	Kim, Kyeongsoon, Kleinman, Hynda K., Lee, Hahn-Jun, Pahan, Kalipada
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5474050/ https://www.ncbi.nlm.nih.gov/pubmed/28623936 http://dx.doi.org/10.1186/s13023-017-0663-8

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