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: 8459259
• 负责人: Mary Schwartz
• 金额: $ 1.5万
• 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/8459259
• 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. PUBLIC HEALTH RELEVANCE: There is currently no effective treatment for Pachyonychia Congenita, an ultra rare blistering skin disorder. A group of scientists and physicians from diverse backgrounds have joined together in the International Pachyonychia Congenita Consortium (IPCC) to identify and develop effective PC treatments. Members of the IPCC have effectively collaborated in a number of small off-label studies, in research to identify effective agents and improve delivery methods and worked together in a Phase 1b FDA-approved clinical trial. The 2013 IPCC meeting will result in a unified collaborative plan to move the most effective research discoveries forward to clinical studies to benefit patients with PC as well as patients with other related disorders of the skin.

描述（由申请人提供）：本赠款申请要求对第十届国际Pachyonychia congenita联盟（IPCC）研讨会的支持，标题为“为Pachyonychia congenita（PC）实施基于研究的临床研究”。这次会议由PC项目（501（c）（3）公共慈善机构）组织和共同赞助，并将于2013年2月14日至17日在犹他州帕克城的Yarrow Hotel（可访问的设施）举行。每一项努力都会 被包括包括代表性不足的科学家/医生，包括妇女，少数民族和学员，以及在儿童健康方面具有专业知识的人。 Pachyonychia congenita（PC）是一种超罕见疾病，任何一个研究中心只有少数患者。 IPCC是一组由75名美国和国际科学家和医生组成的小组，他们同意在治疗PC的治疗剂开发和翻译中共同努力。通过协作努力为PC的研究和临床研究的进步树立了其他罕见疾病的模式，从而为许多其他相关皮肤疾病的努力带来了直接的好处。 通过将来自世界各地的专家召集在一起，2013年研讨会将创造一个强大的环境，以评估完整的研究，激发新的研究并为基于完成的研究的合作临床研究建立前进的道路。除了有关IPCC成员进展的报道外，2013年论坛还将通过包括癌症专家，专家，针对其他皮肤疾病进行研究和临床研究在内的各种领域的专家的意见以及疼痛专家来加强。 IPCC中涉及疼痛专家将有助于对PC患者最重要的领域进步。 已经收到了许多专家的广泛支持，包括E. Birgitte Lane，Pierre Coulombe，Pierre Coulombe，Roger Kaspar，W。H. H. H. H. Irwin McLean，John McGrath，John Milstone，Leonard Milstone，Dennis Roop，Eliecher，Eli Sprecher，Eli Sprecher和其他人认为，该研究对患者的培训是必不可少的。使人衰弱的障碍。 公共卫生相关性：目前尚无对极罕见的泡沫性皮肤疾病的Pachyonychia congenita的有效治疗。来自不同背景的一群科学家和医生共同参与了国际Pachyonychia congenita联盟（IPCC），以识别和开发有效的PC治疗。 IPCC的成员在许多小标签的研究中有效合作，研究识别有效的药物并改善了交付方法，并在1B期FDA批准的临床试验中共同合作。 2013年IPCC会议将制定统一的协作计划，以将最有效的研究发现转向临床研究，以使PC患者以及其他相关疾病的患者受益。