Rafael Holdings宣布尼曼-匹克病C型治疗药物Trappsol® Cyclo™关键三期Transportnpc™研究完成末例患者末次访视

美股速递06-10

Rafael Holdings, Inc. (RFL) 已宣布，其用于治疗尼曼-匹克病C型（NPC）的在研药物Trappsol® Cyclo™，其关键性三期临床试验Transportnpc™研究已完成最后一例患者的最后一次访视。这一里程碑标志着该研究患者参与阶段正式结束，为后续的数据分析及向监管机构提交申请奠定了基础。尼曼-匹克病C型是一种罕见的、进行性且危及生命的遗传性代谢疾病，Trappsol® Cyclo™作为一种潜在的疗法，其临床进展备受关注。

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