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

• Päätekijät: Zufit Hexner-Erlichman (Tekijä), Maria Veiga- (Tekijä), Yoav Zehavi (Tekijä), Zahava Vadasz (Tekijä), Adi D. Sabag (Tekijä), Sameh Tatour (Tekijä), Ronen Spiegel (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: Frontiers Media S.A., 2022-11-01T00:00:00Z.
• Aiheet: article
• Linkit: 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...