UAE批准对2岁及以上儿童的脊椎肌肉萎缩进行基因治疗。 UAE approves gene therapy for spinal muscular atrophy in children age 2 and up.

阿联酋已经批准了Itvisma(onasemnogenne abeparvovec),这是针对两岁以上病人的脊椎肌肉萎缩基因疗法(SMA),成为全球第二个这样做的国家。 The UAE has approved Itvisma (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy (SMA), for patients aged two and older, becoming the second country globally to do so. 阿联酋药品机构(EDE)根据临床证据表明,运动功能持续改善,安全情况良好,因此获得批准。 The Emirates Drug Establishment (EDE) granted approval based on clinical evidence showing sustained motor function improvements and a favorable safety profile. 治疗使用一种与安地诺相关的病毒矢量来取代导致SMA的缺陷基因。 The treatment uses an adeno-associated viral vector to replace the defective gene causing SMA. EDE官员强调了阿联酋高效、科学驱动的监管进程,平衡了速度、严谨度和透明度。 EDE officials highlighted the UAE’s efficient, science-driven regulatory process, balancing speed, rigor, and transparency. 此举支持阿联酋关于可持续、知识驱动的保健系统的愿景,并加强阿联酋作为区域医疗创新枢纽的作用。 The move supports the UAE’s vision for a sustainable, knowledge-driven healthcare system and strengthens its role as a regional hub for medical innovation. 制造商诺华(Norvartis)赞扬这项批准,认为这是向公平获得稀有遗传疾病突破性治疗迈出的一步。 Novartis, the manufacturer, praised the approval as a step toward equitable access to breakthrough therapies for rare genetic diseases.