Effect of empagliflozin treatment on laboratory and clinical findings of patients with glycogen storage disease type Ib: first study from Türkiye.

Summary: Glycogen Storage Disease Type Ib (GSDIb) is a rare genetic condition where the body struggles to manage sugar, leading to dangerously low white blood cell counts and frequent infections. A new study from Türkiye looked at using Empagliflozin—a drug usually prescribed for diabetes—to treat 10 young patients ranging from infants to young adults. The results were highly encouraging: the drug helped boost immune cells, drastically reduced painful mouth sores and skin infections, and kept children out of the hospital. While some side effects like urinary infections occurred, most parents reported a huge leap in quality of life, better sleep, and more active children. It appears to be a promising treatment option for this challenging disease.