Sarepta(SRPT.US)&罗氏(RHHBY.US)DMD基因疗法Elevidys获批扩展适应症

智通财经06-21

智通财经APP获悉，Sarepta Therapeutics(SRPT.US)宣布，美国FDA批准其与罗氏(Roche)(RHHBY.US)联合开发的基因疗法Elevidys(delandistrogene moxeparvovec，SRP-9001)扩展适应症，将年龄至少为4岁的杜氏肌营养不良(DMD)患者纳入其中，这些患者确认带有DMD基因突变。FDA对可行走的患者授予了传统批准。对于不可行走...

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