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.

一个有效的行政结构对发展中国家的设计和执行至关重要。 突触病变联盟（DSC）。行政核心的首要目标是建立一个 环境，在其中相关的科学研究的DSC的目标可以有效和高效地进行 设计、实施和传播。该核心非常成功地建立了一个行政和 在资助的前五年，DSC的管理结构，并与患者密切合作， 倡导团体筹集额外资金，招募受试者和传播有关信息， 三种罕见疾病：TSC、PHTS和PMS。核心将由资深医生/科学家以及 一名生物统计学家、一名项目经理和一名法规经理，他们都具有多中心经验 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，以及DSC所有研究项目的科学和财政监督。的 行政核心将组织调查员启动会议以及执行咨询委员会， 奖学金委员会，指导委员会，研究人员和神经心理学家定期会议 的间隔行政核心将负责以下具体目标：1）协调临床 利用神经心理学的专业知识进行所有三种罕见疾病的研究项目， 生物标志物团队; 2）开发上级核心流程（试点可行性核心和职业提升 核心），满足调查人员和受训人员的需要; 3）充当与其他组织的联络人，包括 PAG、NIH和RDRCN网络; 4）向患者传播DSC相关研究的结果 5）促进一个有凝聚力和一体化的联盟，以最大限度地提高 我们的调查人员和资源的共同努力-这将通过详细的沟通来实现 和业务规划;和6）提供卓越的行政领导方面的管理日- 日常运营，并在整个DSC中保持高质量、高效和具有成本效益的服务。