Bringing Advanced Therapy Medicinal Products (ATMPs) for Parkinson's Disease to the Clinic: The Investigator's Perspective.
Bringing Advanced Therapy Medicinal Products (ATMPs) for Parkinson's Disease to the Clinic: The Investigator's Perspective.
复制标题
将帕金森氏病的晚期治疗药品（ATMP）带到诊所：研究者的观点。
DOI：
10.3233/jpd-212563
复制
发表时间：
2021
期刊：
影响因子：
--
通讯作者：
Daft DM
中科院分区：
文献类型：
--
作者：
Barker RA;Cutting EV;Daft DM
包括细胞和基因治疗在内的先进治疗药物产品（ATMPs）在帕金森病（PD）中的应用令人兴奋。然而，将ATMP用于PD患者的临床试验是复杂的。因此，从研究者的角度来看，在开始这项工作之前问自己两个关键问题是很重要的：首先，你为什么要做这项工作，其次，你是否了解用该产品进行临床试验需要什么。在本文中，我们将简要讨论这两个问题。
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.