Administrative Core - Novel Therapeutics for FSHD

项目摘要 行政核心A将负责规划、发展、协调和 马萨诸塞州大学医学院（UMMS）的管理部门， 面肩肱肌营养不良症（FSHD）。中心领导将继续在查尔斯博士 埃默森在UMMS，路易斯·昆克尔博士在儿童波士顿，凯瑟琳瓦格纳在肯尼迪克里格，所有 经验丰富的研究管理人员和有成就的科学家。核心A的行政单位 将设在UMMS，它有大量的资源，人员和经验的管理， P50中心。核心A将是管理中心所有资源和通信的行政中心， 执行政策和程序，建立一个充满活力的环境，使有效的沟通 以及所有中心成员之间的合作，包括研究者、学员、FSHD患者和 倡导团体，行业合作伙伴，NIH和公众。该中心将通过以下方式促进其使命： 网站，传播中心活动，生物材料和研究的信息，并通过其 与患者倡导团体建立伙伴关系，特别是共同赞助FSHD研究的FSH协会 工作室和研究人员-患者网络会议，以吸引患者，研究人员，学员和NIH 在我们的中心使命。通过核心A，该中心将促进肌肉细胞系的分布， Wellstone FSHD Cell Repository是一种独特的FSHD生物资源。内部委员会将使中心 领导层、调查人员和受训人员有效利用资源，组织内部协商， 交流，并接收关于中心管理、研究进展和培训的反馈。定期- 预定的网络会议将侧重于学员和PI数据共享和研究规划， 一年一度的Wellstone中心务虚会在UMMS将带来调查人员和学员面对面的中心 咨询委员会（CAC）和NIH项目负责人审查研究进展，设定目标并调整中心 美国国家卫生研究院（NIH）的Wellstone中心网络。

PROJECT SUMMARY The Administrative Core A will be responsible for the planning, development, coordination, and overall administration of the University of Massachusetts Medical School (UMMS) W ellstone MD CRC focused on Facioscapulohumeral Muscular Dystrophy (FSHD). Center leadership will continue under Dr. Charles Emerson at UMMS, Dr. Louis Kunkel at Children's Boston, and Kathryn Wagner at Kennedy Krieger, all highly experienced research administrators and accomplished scientists. The administrative unit for Core A will be located at UMMS, which has substantial resources, personnel and experience for management of a P50 Center. Core A will be the administrative hub to manage all Center resources and communications and carry out policies and procedures that establish an energized environment to enable effective communication and collaboration among all the Center constituents, including investigators, trainees, FSHD patients and advocacy groups, industry partners, NIH and the public. The Center will promote its mission through its website, which disseminates information on Center activities, biomaterials and research, and through its partnership with patient advocacy groups, particularly the FSH Society, which co-sponsors FSHD Research W orkshops and Researcher-Patient Networking meetings to engage patients, researchers, trainees and NIH in our Center mission. Through Core A, the Center will promote the distribution of muscle cell lines from its W ellstone FSHD Cell Repository as a unique FSHD bio-resource. Internal committees will enable Center leadership, investigators and trainees to effectively utilize resources, organize internal dataflow and communications, and receive feedback on Center management, research progress and training. Regularly- scheduled web conference meetings will focus on trainee and PI data sharing and research planning, and an annual W ellstone Center Retreat at UMMS will bring investigators and trainees face-to-face with a Center Advisory Committee (CAC) and NIH program leaders to review research progress, set goals and align Center activities with the NIH W ellstone Center Network.