FDA指南:人类神经退行病变的基因治疗(草案)

2021-10-01 美国食品和药品监督管理局 FDA

本指南为申办者提供了针对影响成人和儿童患者的神经退行性疾病开发人类基因治疗 (GT) 产品的建议。 神经退行性疾病是一组异质性疾病,其特征在于中枢神经系统或周围神经系统的结构和功能的进行性退化。 这些

中文标题:

FDA指南:人类神经退行病变的基因治疗(草案)

英文标题:

Human Gene Therapy for Neurodegenerative Diseases

发布日期:

2021-10-01

简要介绍:

本指南为申办者提供了针对影响成人和儿童患者的神经退行性疾病开发人类基因治疗 (GT) 产品的建议。 神经退行性疾病是一组异质性疾病,其特征在于中枢神经系统或周围神经系统的结构和功能的进行性退化。 这些疾病在病因、流行、诊断和治疗方面各不相同,包括遗传和年龄相关疾病。 本指南侧重于产品开发、临床前测试和临床试验设计的考虑。 该指南还讨论了研究性基因治疗产品的上市批准途径。

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    学习了

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