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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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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Kim, Kyeongsoon, Kleinman, Hynda K., Lee, Hahn-Jun, Pahan, Kalipada
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5474050/
https://ncbi.nlm.nih.gov/pubmed/28623936
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0663-8
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