将治疗帕金森病的先进治疗药物（ATMPs）推向临床：研究者的视角。
Bringing Advanced Therapy Medicinal Products (ATMPs) for Parkinson's Disease to the Clinic: The Investigator's Perspective.
机构信息
Department of Clinical Neuroscience, University of Cambridge, Forvie Site, Robinson Way, Cambridge, UK.
MRC-WT Cambridge Stem Cell Institute, Jeffrey Cheah Biomedical Centre Cambridge Biomedical Campus, Puddicombe Way, Cambridge, UK.
出版信息
J Parkinsons Dis. 2021;11(s2):S129-S134. doi: 10.3233/JPD-212563.
Abstract
There is much excitement around the use of advanced therapy medicinal products (ATMPs), including cell and gene treatments, in Parkinson's disease (PD). However, taking an ATMP to clinical trials in patients with PD is complex. As such it is important from an investigator's perspective that they ask themselves two key questions before embarking on such work: firstly, why are you doing it, and, secondly, do you understand what is needed to conduct a clinical trial with that product. In this article, we briefly discuss these two questions.
摘要
在帕金森病（PD）中使用先进治疗药物产品（ATMP），包括细胞和基因治疗，引起了广泛关注。然而，将 ATMP 用于 PD 患者的临床试验非常复杂。因此，从研究者的角度来看，在开展此类工作之前，他们应该问自己两个关键问题：第一，为什么要这样做；第二，是否了解使用该产品进行临床试验所需的条件。本文简要讨论了这两个问题。
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