Grand Challenges in Parkinson's Disease

描述(由申请者提供)：“帕金森氏病的巨大挑战--从分子到人”将重点介绍目前为阻止或延缓帕金森氏病(PD)进展而开发新的干预措施的努力。为了提高帕金森病患者的生活质量，减轻帕金森病患者的经济负担，迫切需要开发帕金森病的疾病修饰疗法。“帕金森病的重大挑战--从分子到人”汇集了基础和临床研究领域的专家，将包括世界级的演讲者，他们将概述有效药物疗法开发的每一步，从识别未得到满足的医疗需求；目标识别和验证；药物筛选和发现，到临床试验设计。我们的目标是：(I)教育受众了解将实验室发现传递给患者所需的多个步骤；(Ii)提供跨学科论坛，讨论目前开发PD疾病修改疗法的方法的局限性；(Iii)与PD患者建立稳固的互动，包括他们作为讲师、讨论者、与会者和组织者的积极参与；(Iv)创造机会，为潜在的合作建立网络和交流想法；以及(V)促进参与者之间的相关讨论。这次会议的目的是在一个有重点的项目中尽可能多地涵盖选定主题的各个方面，聚集该特定主题的顶尖科学家。今年的重点将放在针对帕金森病的治疗方法的开发和与治疗相关的新发现方面已确定的挑战上。该计划包括5个环节：(I)未得到满足的医疗需求；(Ii)帕金森氏病的动物模型；(Iii)靶标发现和确认；(Iv)高通量筛选和药物开发；(V)临床试验设计和结果衡量。这种有重点的方法使我们能够以一种促进互动的形式包括来自不同领域的专家；这些专家通常不会聚集在一起，除非会议形式足够大(例如，世界帕金森大会)。我们的方法确保了研究人员、临床医生、博士后、研究生以及帕金森氏症患者及其家人的参与，在一个友好的环境中保证了高度互动。

DESCRIPTION (provided by applicant): "Grand Challenges in Parkinson's Disease - From Molecules to People" will highlight current efforts to develop novel interventions to stop or retar the progression of Parkinson's disease (PD). The development of disease-modifying therapies for PD is urgently needed to improve the quality of life of people with PD and to reduce the financial burden of PD. "Grand Challenges in Parkinson's Disease - From Molecules to People" gathers experts both from basic and clinical research and will include world-class speakers who will give overviews covering every step in the development of effective drug therapies, from the identification of unmet medical needs; target identification and validation; drug screening and discovery, to clinical trial design. We aim to (i) Educate the audience regarding the multiple steps required to move laboratory discoveries to patients; (ii) Offer a cross-disciplinary forum fo discussions on the limitations of current approaches to the development of disease-modifying therapies for PD; (iii) Establish a solid interaction with people with PD, including their active participation as lecturers, discussants, attendees and organizers; (iv) Create opportunities to network and exchange ideas for potential collaborations; and (v) Promote relevant discussions among the participants. The conference has been designed to cover as many aspects as possible of a selected topic in a focused program, gathering leading scientists in that particular topic. This year the focus will be on the challenges that have been identified in the development of therapies against PD and to novel discoveries of therapeutic relevance. The program consists of 5 sessions, (i) Unmet Medical Needs; (ii) Animal Models of Parkinson's Disease; (iii) Target Discovery And Validation; (iv) High Throughput Screening and Drug Development; (v) Clinical Trial Design And Outcome Measures. This focused approach allows us to encompass experts from different fields in a format that facilitates interactions; these experts would normally not gather unless the conference format is large enough (e.g., World Parkinson's Congress). Our approach has ensured the participation of researchers, clinicians, postdocs, graduate students, as well as of people with PD and their family members, in a welcoming environment that guarantees high degree of interaction.