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

• Váldodahkkit: Zufit Hexner-Erlichman (Dahkki), Maria Veiga- (Dahkki), Yoav Zehavi (Dahkki), Zahava Vadasz (Dahkki), Adi D. Sabag (Dahkki), Sameh Tatour (Dahkki), Ronen Spiegel (Dahkki)
• Materiálatiipa: Girji
• Almmustuhtton: Frontiers Media S.A., 2022-11-01T00:00:00Z.
• Fáttát: article
• Liŋkkat: Connect to this object online.

BackgroundGlycogen 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 additi...