2013 International Pachyonychia Congenita Consortium (IPCC) Symposium

2013年国际先天性厚甲病联盟（IPCC）研讨会

• 批准号: 8636235
• 负责人: Mary Schwartz
• 金额: $ 1.25万
• 依托单位: PACHYONYCHIA CONGENITA PROJECT
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-09-30 至 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8636235
• 关键词: Applications Grants; Area; Bulla; Charities; Child health care; Cities; Clinical Research; Clinical Trials; Development; Disabled Persons; Disease; Ensure; Environment; FDA approved; International; Label; Malignant Neoplasms; Methods; Pain; Patients; Pattern; Phase; Physicians; Positioning Attribute; Progress Reports; Rare Diseases; Request for Applications; Research; Scientist; Specialist; Therapeutic; Translating; Translational Research; Translations; Utah; Woman; Work; Yarrow; base; effective therapy; handicapping condition; improved; kaspar; meetings; member; minority trainee; research study; skin disorder; symposium

DESCRIPTION (provided by applicant): This grant application requests support for the 10th International Pachyonychia Congenita Consortium (IPCC) symposium entitled "Implementing Research-Based Clinical Studies for Pachyonychia Congenita (PC)." This meeting is organized and co-sponsored by PC Project (a 501(c)(3) public charity) and will be held February 14-17, 2013 at the Yarrow Hotel in Park City, Utah (a handicapped accessible facility). Every effort will be made to include underrepresented scientists/physicians, including women, minorities and trainees as well as those with expertise in children's health. Pachyonychia Congenita (PC) is an ultra rare disorder with only a few patients available in any one research center. The IPCC is a group of over 75 US and international scientists and physicians who have agreed to work together in the development and translation of therapeutics to treat PC. Advances in research and clinical studies for PC through collaborative efforts set a pattern for other rare disorders an have a direct benefit to efforts for many other related skin diseases. By bringing experts from around the world together, the 2013 symposium will create a powerful environment to evaluate completed studies, inspire new research and formulate a path forward for cooperative clinical studies based on completed research. In addition to reports of progress from IPCC members, the 2013 forum will be strengthened by input from experts in diverse fields including cancer specialists, experts conducting research and clinical studies for other skin disorders, as well as pain specialists. Involving pain specialists in the IPCC will facilitate progress in this area thatis of highest importance to PC patients. Widespread support for this symposium has been received from many experts including E. Birgitte Lane, Pierre Coulombe, Roger Kaspar, W. H. Irwin McLean, John McGrath, Leonard Milstone, Dennis Roop, Eli Sprecher and others who feel this meeting is essential to translate research discoveries into beneficial treatments for patients As no effective PC treatment is currently available, clinical studies are urgently needed by patients suffering from this debilitating disorder.

描述(申请人提供)：这项拨款申请请求支持第十届国际先天性厚甲联合会(IPCC)研讨会，题为“实施基于研究的临床研究的先天性肥甲症(PC)”。本次会议由PC Project(501(C)(3)公共慈善机构)组织和共同赞助，将于2013年2月14日至17日在犹他州帕克城的亚罗酒店(残疾人无障碍设施)举行。每一次努力都会 应包括代表性不足的科学家/医生，包括妇女、少数群体和受训人员以及在儿童健康方面有专门知识的人。先天性肥甲症(PC)是一种极其罕见的疾病，任何一个研究中心都只有少数患者可用。IPCC是一个由超过75名美国和国际科学家和医生组成的团体，他们同意在治疗PC的疗法的开发和翻译方面合作。通过合作努力，PC的研究和临床研究的进展为其他罕见疾病设定了模式，并对许多其他相关皮肤病的努力有直接好处。通过将来自世界各地的专家聚集在一起，2013年研讨会将创造一个强大的环境，以评估已完成的研究，激励新的研究，并制定一条基于已完成研究的合作临床研究的前进道路。除了IPCC成员的进展报告外，2013年论坛还将得到不同领域专家的意见加强，包括癌症专家、进行其他皮肤病研究和临床研究的专家以及疼痛专家。让疼痛专家参与IPCC将促进这一领域的进展，这对PC患者来说是最重要的。这次研讨会得到了许多专家的广泛支持，包括E.Birgitte Lane，Pierre Coulombe，Roger Kaspar，W.H.Irwin McLean，John McGrath，Leonard Milstone，Dennis Roop，Eli Sprecher和其他人，他们认为这次会议对于将研究发现转化为对患者有益的治疗至关重要，因为目前没有有效的PC治疗方法，患有这种衰弱障碍的患者迫切需要临床研究。