Background and aims: Hypoglycemia is a typical, potentially life-threatening symptom in classical galactosemia with blood galactose considerably elevated (up to ∼100 mg/dl). Therefore we tested ...

It also significantly reduced plasma galactitol levels in adults and children with galactosemia. Additional studies provided strong efficacy and safety data across 185 classic galactosemia ...

Additionally, the company has filed a marketing authorization application (MAA) with the European Medicines Agency (EMA) to treat classic galactosemia. The MAA was validated by the EMA in December ...