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Cholbam® and Zellweger spectrum disorders: treatment implementation and management

BACKGROUND: Zellweger spectrum disorders (ZSDs) are a rare, heterogenous group of autosomal recessively inherited disorders characterized by reduced peroxisomes numbers, impaired peroxisomal formation, and/or defective peroxisomal functioning. In the absence of functional peroxisomes, bile acid synt...

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Detalles Bibliográficos
Autores principales: Anderson, Janaina Nogueira, Ammous, Zineb, Eroglu, Yasemen, Hernandez, Erick, Heubi, James, Himes, Ryan, Palle, Sirish
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8439061/
https://www.ncbi.nlm.nih.gov/pubmed/34521419
http://dx.doi.org/10.1186/s13023-021-01940-z

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