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

• 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
• 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

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(申请人提供)：骨骼健康对整体健康和生活质量至关重要；骨骼系统提供身体活动、支持和保护，并是基本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。到目前为止，已描述了近500种独特的罕见骨病，这些疾病总共影响到5000名活产儿中的1名，占所有出生缺陷的5%。通常情况下，这些疾病造成的残疾会影响个人的一生。目前，这些疾病中的绝大多数都没有治愈方法，但对罕见骨病的研究有助于阐明生物途径，揭示有价值的药物发现目标，也使常见骨病患者受益。这项拨款申请申请部分资助由美国国家骨健康联盟(NBHA)与罕见骨病患者网络合作，并得到美国骨与矿物研究学会(ASBMR)和美国骨与关节倡议(USBJI)支持的研讨会“从罕见骨病研究中学到的骨骼生物学的机械学和治疗学见解”。研讨会的具体目的是：1)召集一个包括研究人员、临床医生和罕见骨病患者在内的多学科专家小组，以增进我们对骨骼生物学的了解，并鼓励开发新的治疗方法，以改善患有常见和罕见骨病的患者的预后；2)找出我们对罕见骨病潜在生物机制的认识差距，并在翻译和治疗领域分享有效的研究战略；3)通过在ASBMR 2014年年会的前一天举行活动并为大量年轻研究人员提供资金参加，鼓励广泛参与；以及4)通过在同行评议的期刊上发表该项目的会议记录来广泛传播这些结果。为期一天的研讨会将于2014年9月11日在德克萨斯州休斯敦举行，也就是ASBMR 2014年会的前一天，该年会是骨和矿物研究领域学术和临床领袖的主要聚会。这一配对将鼓励最多的科学家、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演讲，以及启动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，并将确保纳入妇女、残疾个人和代表人数不足的群体的参与者。

项目成果

Mechanistic and therapeutic insights gained from studying rare skeletal diseases.

从研究罕见骨骼疾病中获得的机制和治疗见解。

• DOI: 10.1016/j.bone.2015.03.016
• 发表时间: 2015
• 期刊: Bone
• 影响因子: 4.1
• 作者: Tosi,LauraL;Warman,MatthewL
• 通讯作者: Warman,MatthewL