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Diagnosing neuronopathic Gaucher disease: New considerations and challenges in assigning Gaucher phenotypes

Gaucher disease (GD), resulting from biallelic mutations in the gene GBA1, is a monogenic recessively inherited Mendelian disorder with a wide range of phenotypic presentations. The more severe forms of the disease, acute neuronopathic GD (GD2) and chronic neuronopathic GD (GD3), also have a continu...

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Bibliografiska uppgifter
I publikationen:Mol Genet Metab
Huvudupphovsmän: Daykin, Emily, Ryan, Emory, Sidransky, Ellen
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2021
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7884077/
https://ncbi.nlm.nih.gov/pubmed/33483255
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2021.01.002
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