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Exploratory study of autophagy inducer sirolimus for childhood cerebral adrenoleukodystrophy

OBJECTIVES: X-linked adrenoleukodystrophy (ALD) is a peroxisomal disease caused by mutations in the ABCD1 gene. Childhood cerebral ALD (CCALD) is characterized by inflammatory demyelination, rapidly progressing, often fatal. Hematopoietic stem cell transplant only delays disease progression in patie...

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Detalles Bibliográficos
Autores principales:	Luo, Xiao-Mei, Liu, Li-Ying, Wang, Qiu-Hong, Wang, Yang-Yang, Wang, Jing, Yang, Xiao-Yan, Li, Shi-Jun, Zou, Li-Ping
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Pediatrics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10289280/ https://www.ncbi.nlm.nih.gov/pubmed/37360358 http://dx.doi.org/10.3389/fped.2023.1187078

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