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Favorable outcome of empagliflozin treatment in two pediatric glycogen storage disease type 1b patients

BACKGROUND: Glycogen storage disease type 1b (GSD1b) is an ultra-rare autosomal recessive disorder, caused by mutations in SLC37A4 gene. Affected patients present with episodes of fasting hypoglycemia and lactic acidosis, hepatomegaly, growth retardation, hyperlipidemia and renal impairment. In addi...

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Detalles Bibliográficos
Autores principales: Hexner-Erlichman, Zufit, Veiga-da-Cunha, Maria, Zehavi, Yoav, Vadasz, Zahava, Sabag, Adi D., Tatour, Sameh, Spiegel, Ronen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9727171/
https://www.ncbi.nlm.nih.gov/pubmed/36507137
http://dx.doi.org/10.3389/fped.2022.1071464