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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...
Autores principales: | Anderson, Janaina Nogueira, Ammous, Zineb, Eroglu, Yasemen, Hernandez, Erick, Heubi, James, Himes, Ryan, Palle, Sirish |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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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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