Developmental Synaptopathies Associated with TSC, PTEN and SHANK3 Mutations Administrative Core

一个完善的ffi管理结构对开发项目的设计和实施至关重要 突触病态联盟(DSC)。行政核心的首要目标是创建一个 与数字科学委员会的目标相关的科学研究能够有效和高效地开展的环境 设计、执行和传播。这个核心非常成功地建立了一个管理和 在资助的头五年中，DSC的管理结构，并与患者密切合作 倡导团体筹集额外资金，招募研究对象，并传播关于 三种罕见疾病：TSC、PHTS和PMS。核心中心将配备高级医生/科学家以及 一名生物统计师、一名项目经理和一名监管经理，他们都有多个地点的经验 临床研究项目。行政核心侧重于促进沟通、协作、 规划、数据共享和科学fic和fi规模监督所有研究项目的数字安全委员会。这个 行政核心将组织调查员启动会议以及执行咨询委员会， 定期召开学术奖励委员会、指导委员会、研究人员和神经心理学家会议 间隔时间。管理核心将负责以下具体fi目标：1)协调临床 所有三种罕见疾病的研究项目都利用了神经心理学和 生物标记团队；2)开发卓越的核心流程(试点可行性核心和职业提升 核心)，满足调查员和受训人员的需要；3)作为与其他组织的联络人，包括 PAGS、NIH和RDRCN网络；4)向患者传播DSC相关研究的结果 和调查员社区；5)促进一个有凝聚力和一体化的联合体，以最大限度地 我们的调查人员和资源的共同努力-这将通过详细的沟通实现 和业务规划；以及6)在管理日常工作方面提供出色的行政领导- 维持日常运作，并在整个DSC维持高质量、高效率和高成本效益的服务。

An efficient administrative structure is essential to the design and implementation of the Developmental Synaptopathies Consortium (DSC). The overarching goal of the Administrative Core is to create an environment in which scientific research relevant to the goals of the DSC can be effectively and efficiently designed, conducted and disseminated. This Core was very successful in establishing an administrative and management structure for the DSC in the first five years of funding and has worked closely with patient advocacy groups to raise additional funds, recruit subjects and disseminate information with respect to the three rare disorders: TSC, PHTS and PMS. The Core will be staffed by senior physician/scientists as well as a biostatistician, a project manager and a regulatory manager, all of whom are experienced in multi-site clinical research projects. The Administrative Core focuses on facilitating communication, collaboration, planning, data sharing, and scientific and fiscal oversight of all research projects for the DSC. The Administrative Core will organize the investigator launch meeting as well as the Executive Advisory Committee, Scholarly Awards Committee, Steering Committee, study staff and neuropsychologist meetings at regular intervals. The Administrative Core will be responsible for the following Specific Aims: 1) coordinating clinical research projects across all three rare disorders leveraging the expertise of the neuropsychology and biomarker teams; 2) developing superior core processes (Pilot Feasibility Core and Career Enhancement Core), meeting the needs of investigators and trainees; 3) serving as a liaison to other organizations, including PAGs, the NIH, and the RDRCN Network; 4) disseminating the results of DSC-related research to the patient and investigator communities; 5) promoting a cohesive and integrated consortium in order to maximize the combined efforts of our investigators and resources – which will be achieved through detailed communication and operational planning; and 6) providing outstanding administrative leadership with respect to managing day- to-day operations and maintaining high-quality, efficient and cost-effective services throughout the DSC.