UniQure's AMT-191 gene therapy for Fabry disease receives Orphan Drug Designation from FDA.

UniQure has received Orphan Drug Designation from the FDA for AMT-191, its investigational gene therapy aimed at treating Fabry disease, a rare genetic disorder. This designation follows the first patient dosing in a Phase I/IIa trial. The recognition highlights the potential of AMT-191 to provide significant benefits for patients suffering from Fabry disease.

September 23, 2024
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