The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 years of age) than the children treated in previous clinical studies 1-6 Nearly all patients treated maintained or improved motor milestones after 52 weeks, with most switching to the one-time gene therapy from another chronically administered disease-modifying therapy 1-6The SMART study is the first open-label clinical study of Zolgensma to include previously treated patients 1-6 Basel, March 4, 2024 – Novartis today presented new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA).

SMART研究补充了越来越多的证据，证明在年龄较大且体重较重（1.5-9.1岁）的患者群体中使用唑根斯玛比在以前的临床研究中治疗的儿童1-6几乎所有接受治疗的患者在52周后都保持或改善了运动里程碑，随着大多数患者从另一种长期服用的疾病缓解疗法1-6转向一次性基因疗法，SMART研究是佐根斯玛的第一项开放标签临床研究，包括先前接受治疗的患者1-6巴塞尔，2024年3月4日-诺华今天提供了新数据，继续支持佐根斯玛®（onasemnogene abeparvovec）的临床益处，唯一用于治疗脊髓性肌萎缩症（SMA）的一次性基因疗法。

Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment. The new clinical results supplement emerging real-world experience and use of this innovative therapy in older and heavier children in countries where authorized use is not restricted by age.

SMART研究的最终数据突出了Zolgensma在SMA体重≥8.5 kg至≤21 kg的儿童中的安全性和有效性，平均年龄为4.69岁，其中大多数（21/24，87.5%）在治疗时停止使用另一种疾病缓解疗法。在授权使用不受年龄限制的国家，新的临床结果补充了新兴的现实世界经验以及这种创新疗法在年龄较大和体重较大的儿童中的使用。

These data are among a Zolgensma data set being presented during the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida, March 3 – 6. “The results from the SMART study provide evidence that Zolgensma is clinically beneficial for older and heavier patients with SMA, many of whom have had prior treatment with another disease-modifying therapy,” said Dr.

这些数据是3月3日至6日在佛罗里达州奥兰多举行的2024年肌肉萎缩症协会（MDA）临床和科学会议上提出的Zolgensma数据集之一。“SMART研究的结果提供了证据，证明佐根斯玛对老年和体重较重的SMA患者具有临床益处，其中许多患者曾接受过另一种疾病缓解疗法的治疗，”Dr。

Hugh McMillan, Pediatric Neurologist. “These data inform the use of Zolgensma in children up to 21 kg, supporting the use of a one-time gene replacement therapy as a therapeutic opt.

休·麦克米兰，儿科神经病学家。“这些数据表明，Zolgensma可用于21公斤以下的儿童，支持使用一次性基因替代疗法作为治疗选择。