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

• Príomhchruthaitheoirí: Zufit Hexner-Erlichman (Údar), Maria Veiga- (Údar), Yoav Zehavi (Údar), Zahava Vadasz (Údar), Adi D. Sabag (Údar), Sameh Tatour (Údar), Ronen Spiegel (Údar)
• Formáid: LEABHAR
• Foilsithe / Cruthaithe: Frontiers Media S.A., 2022-11-01T00:00:00Z.
• Ábhair: article
• Rochtain ar líne: 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...