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2-methylacetoacetyl-coenzyme A thiolase (beta-ketothiolase) deficiency: one disease - two pathways

BACKGROUND: 2-methylacetoacetyl-coenzyme A thiolase deficiency (MATD; deficiency of mitochondrial acetoacetyl-coenzyme A thiolase T2/ “beta-ketothiolase”) is an autosomal recessive disorder of ketone body utilization and isoleucine degradation due to mutations in ACAT1. METHODS: We performed a syste...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Grünert, Sarah C., Sass, Jörn Oliver
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7187484/
https://ncbi.nlm.nih.gov/pubmed/32345314
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01357-0
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