Niemann–Pick double win

IF 33.1 1区生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Nature biotechnology Pub Date : 2024-10-14 DOI:10.1038/s41587-024-02450-6

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尼曼-皮克双赢

一周之内，美国食品和药物管理局批准了两种治疗 C 型尼曼-皮克病（NPC）的药物。其中一种药物是 Zevra Therapeutics 公司的 Miplyffa（arimoclomol），它是 FDA 批准的第一种与酶抑制剂 miglustat 联用治疗 NPC 相关神经症状的药物。另一种药物是 IntraBio 的 Aqneursa（左旋乙酰亮氨酸），这是一种口服改性氨基酸（N-乙酰-左旋亮氨酸）产品，可增强脑葡萄糖代谢，重振线粒体 ATP 相关功能。鼻咽癌是由 NPC1 或 NPC2 基因突变引起的，这种突变会导致溶酶体贮存异常，影响细胞中胆固醇和脂质的转运，从而导致进行性神经功能损害和器官功能障碍。鼻咽癌患者的平均寿命约为 13 年，直到现在，这种罕见疾病还没有获得批准的治疗方法。

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来源期刊

Nature biotechnology 工程技术-生物工程与应用微生物

CiteScore

63.00

自引率

1.70%

发文量

382

审稿时长

3 months

期刊介绍： Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.

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