Pseudoxanthoma Elasticum Research 2010 Conference

弹性假黄瘤研究 2010 年会议

• 批准号: 8006489
• 负责人: SHARON F. TERRY
• 金额: $ 3.8万
• 依托单位: PXE INTERNATIONAL, INC.
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-07-15 至 2011-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8006489
• 关键词: Applications Grants; Attention; Basic Science; Biological Markers; Biology; Blindness; Cardiology; Cardiovascular Diseases; Clinical; Clinical Investigator; Clinical Sciences; Clinical Trials; Communities; Consensus; Data Collection; Dermatology; Diagnostic; Disabled Persons; Discipline; Disease; Fertilization; Foundations; Future; Gastroenterology; General Population; Genetic Predisposition to Disease; Individual; International; Metabolic; Metabolism; Modeling; Molecular Genetics; Ophthalmology; Pathogenesis; Patient Care; Physicians; Preparation; Problem Solving; Pseudoxanthoma Elasticum; Public Health; Request for Applications; Research; Research Personnel; Scientist; Skin Wrinkling; Solutions; Therapeutic Intervention; Translational Research; Vitamin K; base; clinical care; genetic epidemiology; handicapping condition; interest; meetings; symposium

DESCRIPTION (provided by applicant): This grant application requests partial support for a Conference, entitled "PXE Research 2010". The meeting will be held September 8 and 9th, 2010 at the Bethesda North Marriott, Bethesda, MD, a handicapped accessible hotel. The meeting is coordinated and co-sponsored by PXE International, a nonprofit foundation, established in 1995, to advance research on pseudoxanthoma elasticum (PXE). This conference will capitalize on significant progress made in basic understanding of the genetics, etiology and potential treatments for the disease since the last research meeting in 2004. Though these advances are significant, they have highlighted a lack of linkage between the basic genetic and molecular advances and the pathogenesis of this disease. The specific aim of this Conference is to review the current status of basic and translational research on PXE, determine the most relevant biomarkers and clinical endpoints in preparation for clinical trials, and evaluate a number of hypothetical therapeutic interventions including vitamin K therapy and anti-angiogensis therapies. Additionally, the meeting will review the understanding of the clinical manifestations of PXE, to come to consensus on diagnostic criteria. The Conference will provide a forum for investigators to discuss relevant advances in transporter biology, metabolism, genetics, and epidemiology and for clinically oriented colleagues and PXE International to jointly plan for future research and translational applications. The audience is expected to represent a broad spectrum of individuals with varying expertise including practicing clinicians caring for patients with PXE, physician-scientists and basic science investigators and lay leaders, all of whom share an interest in PXE. Further, the Conference will provide a forum for a cross-fertilization of ideas between the basic and clinical sciences that may not be achievable in more general meetings, and that is essential to translational research. The presence of clinical investigators from the disciplines of dermatology, ophthalmology, gastroenterology, metabolics, and cardiology will allow broad-based discussion of PXE with its many systemic effects, to inform the basic science to focus it, and conversely to set best practice standards for data collection and clinical care. A research plan will be developed and disseminated. Special attention is paid to the involvement of young investigators and trainees in this meeting, and involving both scientists and clinicians from underrepresented communities.

描述（由申请人提供）：此资助申请要求对一个名为“PXE研究2010”的会议提供部分支持。会议将于2010年9月8日和9日在马里兰州贝塞斯达北万豪酒店举行，这是一家残疾人无障碍酒店。该会议由PXE国际组织协调和共同赞助，该组织是一个非营利基金会，成立于1995年，旨在推进弹性假黄瘤（PXE）的研究。本次会议将利用自2004年上次研究会议以来在遗传学，病因学和潜在治疗方法的基本理解方面取得的重大进展。虽然这些进展是重要的，但它们突出了基本遗传和分子进展与这种疾病的发病机制之间缺乏联系。本次会议的具体目的是审查PXE的基础和转化研究的现状，确定最相关的生物标志物和临床终点，为临床试验做准备，并评估一些假设的治疗干预措施，包括维生素K治疗和抗血管生成治疗。此外，会议将审查对PXE临床表现的理解，以就诊断标准达成共识。会议将为研究人员提供一个论坛，讨论转运蛋白生物学，代谢，遗传学和流行病学的相关进展，并为临床导向的同事和PXE国际共同规划未来的研究和翻译应用。观众预计将代表广泛的个人与不同的专业知识，包括执业临床医生照顾病人与PXE，医生，科学家和基础科学研究人员和外行领导人，所有人都有兴趣在PXE。此外，会议将为基础科学和临床科学之间的思想交流提供一个论坛，这在更一般的会议中可能无法实现，这对转化研究至关重要。来自皮肤病学、眼科学、胃肠病学、代谢学和心脏病学等学科的临床研究人员的存在将允许对PXE及其许多全身效应进行广泛的讨论，以告知基础科学将其作为重点，并相反地为数据收集和临床护理设定最佳实践标准。将制定和传播一项研究计划。会议特别注意青年调查人员和受训人员的参与，并邀请代表性不足的社区的科学家和临床医生参加。