用于罕见和超罕见遗传性神经疾病的反义药物。

Antisense drugs for rare and ultra-rare genetic neurological diseases.

机构信息

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

出版信息

Neuron. 2023 Aug 16;111(16):2465-2468. doi: 10.1016/j.neuron.2023.05.027. Epub 2023 Jun 23.

DOI:10.1016/j.neuron.2023.05.027

Abstract

The regulatory approvals of nusinersen and tofersen, plus the large body of clinical and preclinical data from other drugs, have significantly de-risked antisense technology for neurological diseases. The platform learnings over the last 2 decades can be applied to subsequent drugs to improve the efficiency of discovering effective neuro-therapeutics.

摘要

监管部门批准了nusinersen 和 tofersen 的上市，加上其他药物的大量临床前和临床数据，这使得反义技术在神经疾病领域的风险大大降低。过去 20 年的平台经验可以应用于后续药物，以提高发现有效神经疗法的效率。