uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
AMTAmerican Tower(AMT) GlobeNewswire News Room·2024-09-23 19:05

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-ce ...