The effect of the glucosylceramide synthase inhibitor lucerastat on cardiac repolarization: results from a thorough QT study in healthy subjects

BACKGROUND: Fabry disease is a rare inherited glycosphingolipid storage disorder caused by deleterious mutations in the GLA gene coding for the lysosomal enzyme α-galactosidase A. The glucosylceramide synthase inhibitor lucerastat is an iminosugar with potential to provide oral substrate reduction t...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Principais autores: Mueller, Markus S., Sidharta, Patricia N., Voors-Pette, Christine, Darpo, Borje, Xue, Hongqi, Dingemanse, Jasper
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2020
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7590462/
https://ncbi.nlm.nih.gov/pubmed/33109218
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01582-7
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