针对法布里病的UniQure的AMT-191基因疗法获得FDA的孤儿药物指标. UniQure's AMT-191 gene therapy for Fabry disease receives Orphan Drug Designation from FDA.

UniQure已获得FDA对AMT-191的孤儿药物指定,该药物用于治疗罕见遗传疾病法布里病. 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. 这一指定是在第一阶段/IIa试验中第一位病人服药之后作出的。 This designation follows the first patient dosing in a Phase I/IIa trial. 承认强调了AMT-191号协议为患有法布里病的病人提供重大福利的潜力。 The recognition highlights the potential of AMT-191 to provide significant benefits for patients suffering from Fabry disease.