Mechanistic and Therapeutic Insights into Skeletal Biology Learned from the Study

从研究中获得的骨骼生物学机制和治疗见解

基本信息

批准号：
8720467
负责人：
Matthew L Warman
金额：
$ 2.5万
依托单位：
NATIONAL OSTEOPOROSIS FOUNDATION
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-06-01 至 2015-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8720467
关键词：
Affect Age American Applications Grants Area Baltimore Basic Science Biological Biology Bone Diseases Cells Clinical Clinical Research Collaborations Congenital Abnormality Development Disease Disease Progression Educational workshop Ensure Event Family Fostering Funding Genes Genetic Goals Growth Health Health Alliance Human Individual Industry Joints Journals Knowledge Learning Live Birth Maintenance Maryland Minerals Osteoporosis Outcome Participant Pathway interactions Patients Pattern Peer Review Publications Publishing Quality of life Rare Diseases Reporting Request for Applications Research Research Personnel Scientist Skeletal system Skeleton Societies Surgeon Texas Therapeutic Translational Research Underrepresented Minority United States United States National Institutes of Health Update Woman abstracting bone bone health bone mass design disability drug discovery improved insight lectures meetings novel prevent programs public health relevance skeletal symposium

项目摘要

DESCRIPTION (provided by applicant): Bone health is critical to overall health and quality of life; the skeletal system provides the body mobility, support and protection, and serves as a major reservoir for essential minerals. But not everyone enjoys good bone health. To date, nearly 500 unique rare bone diseases have been described, which in the aggregate affect 1 in 5,000 live births, and comprise 5% of all birth defects. Typically the disability caused by these diseases affects individuals throughout their lives. Currently, the vast majority of these disorder have no cure, yet studying rare bone diseases has helped elucidate biological pathways and reveal valuable targets for drug discovery that are benefitting patients with common bone diseases too. This grant application requests partial support for "Mechanistic and Therapeutic Insights into Skeletal Biology Learned from the Study of Rare Bone Diseases," a workshop being coordinated by the National Bone Health Alliance (NBHA) in partnership with the Rare Bone Disease Patient Network and with the support of the American Society for Bone and Mineral Research (ASBMR) and the United States Bone and Joint Initiative (USBJI). The specific aims of the workshop are to: 1) Convene a multi-disciplinary group of experts including researchers, clinicians, and patients with rare bone diseases to advance our understanding of skeletal biology and encourage the development of novel therapies to improve outcomes for individuals with both common and rare bone diseases; 2) Identify gaps in our knowledge regarding the biologic mechanisms underlying rare bone disorders and share effective research strategies in translational and therapeutic areas; 3) Encourage broad participation both by holding the event the day before the ASBMR 2014 Annual Meeting and providing funding for a significant number of young investigators to attend; and 4) Disseminate the results broadly by publishing the proceedings of the program in a peer-reviewed journal. The one-day workshop will be held on September 11, 2014 in Houston, Texas, the day before the ASBMR 2014 Annual Meeting, the premier gathering of academic and clinical leaders in bone and mineral research. This pairing will encourage the greatest number of scientists, clinicians, and young investigators to participate in the workshop. The workshop will include lectures by invited speakers, and multiple opportunities to initiate collaborations and encourage future research partnerships. The planning committee has been confirmed and will ensure inclusion of women, individuals with disabilities, and participants from underrepresented groups.

描述（由申请人提供）：骨骼健康对于整体健康和生活质量至关重要；骨骼系统提供身体的活动性、支撑和保护，并作为必需矿物质的主要储存库。但并不是每个人都享有良好的骨骼健康。迄今为止，已有近 500 种独特的罕见骨病被描述，这些疾病总共影响五千分之一的活产儿，占所有出生缺陷的 5%。通常，这些疾病引起的残疾会影响个人的一生。目前，绝大多数此类疾病无法治愈，但研究罕见骨病有助于阐明生物学途径，并揭示有价值的药物发现靶点，这些靶点也使常见骨病患者受益。本拨款申请要求部分支持“从罕见骨病研究中获得的骨骼生物学机制和治疗见解”，该研讨会由国家骨健康联盟 (NBHA) 与罕见骨病患者网络和罕见骨病患者网络合作协调举办。美国骨与矿物质研究学会 (ASBMR) 和美国骨与关节倡议 (USBJI) 的支持。研讨会的具体目标是： 1) 召集一个由研究人员、临床医生和罕见骨病患者组成的多学科专家组，以增进我们对骨骼生物学的理解，并鼓励开发新疗法，以改善罕见骨病患者的治疗结果常见和罕见的骨病； 2) 找出我们在罕见骨病生物学机制方面的知识差距，并分享转化和治疗领域的有效研究策略； 3) 通过在ASBMR 2014年会前一天举办活动并为大量年轻研究者参加提供资金来鼓励广泛参与； 4) 通过在同行评审期刊上发表该项目的会议记录来广泛传播结果。为期一天的研讨会将于 2014 年 9 月 11 日在德克萨斯州休斯顿举行，即 ASBMR 2014 年年会的前一天，这是骨和矿物研究领域学术和临床领导者的重要聚会。这种配对将鼓励尽可能多的科学家、临床医生和年轻研究人员参加研讨会。研讨会将包括受邀演讲者的讲座，以及发起合作和鼓励未来研究伙伴关系的多种机会。规划委员会已经确定，并将确保妇女、残疾人和代表性不足群体的参与者的参与。