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Novel ALDH5A1 variants and genotype: Phenotype correlation in SSADH deficiency

OBJECTIVE: To determine genotype–phenotype correlation in succinic semialdehyde dehydrogenase (SSADH) deficiency. METHODS: ALDH5A1 variants were studied with phenotype correlation in the SSADH natural history study. Assignment of gene variant pathogenicity was based on in silico testing and in vitro...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: DiBacco, Melissa L., Pop, Ana, Salomons, Gajja S., Hanson, Ellen, Roullet, Jean-Baptiste, Gibson, K. Michael, Pearl, Phillip L.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7713737/
https://ncbi.nlm.nih.gov/pubmed/32887777
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000010730
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