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

与 TSC、PTEN 和 SHANK3 突变相关的发育性突触病管理核心

• 批准号: 10701738
• 负责人: MUSTAFA SAHIN
• 金额: $ 46.43万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-20 至 2024-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10701738
• 关键词: Access to Information; Administrative Efficiency; Advisory Committees; Award; Biological Markers; Cell Nucleus; Clinical; Clinical Practice Guideline; Clinical Research; Collaborations; Communication; Communities; Data Coordinating Center; Development; Disease; Enrollment; Ensure; Environment; Funding; Goals; Good Clinical Practice; Grant; Health Professional; Leadership; Mutation; Neuropsychology; PTEN gene; Patients; Physicians; Pilot Projects; Policies; Procedures; Process; Rare Diseases; Research; Research Personnel; Research Project Grants; Resources; Scientist; Services; Site; Structure; Sum; System; Training; Travel; United States National Institutes of Health; Work; career; clinical research site; cost effective; data sharing; design; experience; flexibility; meetings; operation; patient advocacy group; recruit; research study

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.

有效的行政结构对于设计和实施发展至关重要 突触疗法财团（DSC）。行政核心的总体目标是创建一个 与DSC目标相关的科学研究的环境可以有效，有效 设计，进行和传播。该核心在建立行政和 DSC在最初五年的资金中的管理结构，并与患者密切合作 倡导团体筹集额外资金，招募主题并传播有关 三种罕见疾病：TSC，PHT和PMS。核心将由高级物理学/科学家以及 生物统计学家，项目经理和监管经理，所有这些人都经验丰富 临床研究项目。行政核心重点是促进沟通，协作， 对DSC的所有研究项目的计划，数据共享以及科学和规模的监督。 行政核心将组织调查员启动会议以及执行咨询委员会 学术奖项委员会，指导委员会，研究人员和神经心理学家会议定期 间隔。行政核心将负责以下特定目的：1）协调临床 所有三种罕见疾病的研究项目都利用神经心理学的专业知识和 生物标志物团队； 2）开发出色的核心流程（飞行员可行性核心和职业增强 核心），满足调查人员和学员的需求； 3）作为与其他组织的联络员，包括 PAGS，NIH和RDRCN网络； 4）向患者传播与DSC相关研究的结果 和调查员社区； 5）促进一个凝聚力和综合财团，以最大化 我们的调查人员和资源的共同努力 - 将通过详细的沟通来实现 和运营计划； 6）在管理日期方面提供出色的行政领导 - 今天的DSC中，今天的运营以及维持高质量，高效和成本效益的服务。