STAT+：诺华提及此前未知的肿瘤病例，可能与其SMA基因疗法相关
摘要
诺华已确认七例此前未知的病例，这些病例是在接受其脊髓性肌萎缩症治疗的儿童中发现了肿瘤，这一令人意外的进展引发了家庭、医生和患者权益组织的担忧。这些病例目前仅被视为潜在的安全性信号，于周四由一家权益组织和一个Facebook页面公布，此前诺华于本周早些时候向神经科医生披露了这一信息。一家名为SMA Europe的权益组织发表声明称，监管机构目前正在审查这些病例。该制药公司发表了自身声明，称自其名为Zolgensma的治疗药物于2019年获批以来，已收到九份关于既往接受治疗患者出现肿瘤的报告。其中
English
Novartis has acknowledged seven previously unknown instances in which tumors were found in children who were treated with its spinal muscular atrophy therapy, a surprising development that has raised concerns among families, physicians, and patient advocacy groups. The cases, which for the moment are being seen as only a potential safety signal, were publicized on Thursday by an advocacy group and on a Facebook page after Novartis earlier this week disclosed the information to neurologists. One advocacy group, SMA Europe, issued a statement saying that regulatory authorities are now examining the cases. The drugmaker issued its own statement saying that since its treatment — called Zolgensma — was approved in 2019, it has received nine reports of tumors in previously treated patients. Of t