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

• Main Authors: Zufit Hexner-Erlichman (Author), Maria Veiga- (Author), Yoav Zehavi (Author), Zahava Vadasz (Author), Adi D. Sabag (Author), Sameh Tatour (Author), Ronen Spiegel (Author)
• Format: Knjiga
• Izdano: Frontiers Media S.A., 2022-11-01T00:00:00Z.
• Teme: article
• Online dostop: 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...