IPCC Scientific Meeting

IPCC科学会议

• 批准号: 7113902
• 负责人: Mary E Schwartz
• 金额: $ 5万
• 依托单位: PACHYONYCHIA CONGENITA PROJECT
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2006-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7113902
• 关键词: autosomal dominant trait; congenital disorders; meeting /conference /symposium; skin derivative; skin disorder; travel

DESCRIPTION (provided by applicant): This grant application requests partial support for a Symposium entitled "Reaching Clinical Trials for Pachyonychia Congenita." The meeting will be held May 2 and 3, 2006 at the Philadelphia Marriott, Philadelphia, PA, a handicapped accessible hotel. The meeting is organized and co-sponsored by PC Project, a nonprofit public charity, established in 2003 dedicated to the development and delivery of therapeutics to treat PC. Prior scientific meetings sponsored by PC Project were held in 2004 and 2005. Those meetings resulted in: An active collaboration of 25 researchers organized as the International PC Consortium (IPCC). A comprehensive PC research plan which focuses on the most promising technologies, prevents duplication of efforts, and enhances sharing of resources. Publication of a Symposium Proceedings published by the Journal of Investigative Dermatology. An IRB-patient registry, a comprehensive full-access PC bibliography. Formation of the International PC Physicians Network which serves as an adjunct to the IPCC for delivery of patient care. The goal of the 2006 meeting is to establish the steps and structure necessary for effective PC clinical trials utilizing delivery technology and agents which are now available. The meeting will create a forum for discussion of research advances in gene-based therapy for PC and the development and use of model systems including development of a PC mouse. In addition to reports of progress from IPCC members, this forum will be strengthened by input from experts in diverse fields including scientists and clinicians seeking clinical trials for other rare disorders. The FDA/OOPD will also participate in the meeting to provide expertise in effectively utilizing the rare drug designation. The meeting will also involve contributions from junior investigators and a special effort will be made to include women and minorities in the discussion groups and presentations.

描述（由申请人提供）：本拨款申请请求为题为“先天性厚甲症临床试验”的研讨会提供部分支持。会议将于 2006 年 5 月 2 日至 3 日在宾夕法尼亚州费城的费城万豪酒店（一家残疾人专用酒店）举行。该会议由 PC Project 组织和共同赞助，PC Project 是一家非营利性公共慈善机构，成立于 2003 年，致力于开发和提供治疗 PC 的疗法。 PC Project 赞助的先前科学会议于 2004 年和 2005 年举行。 这些会议的结果是： 25 名研究人员积极合作，组建了国际 PC 联盟 (IPCC)。 全面的 PC 研究计划，重点关注最有前途的技术，防止重复工作，并加强资源共享。 出版《研究皮肤病学杂志》出版的研讨会论文集。 IRB 患者登记册、全面的、可完全访问的 PC 参考书目。 成立国际 PC 医生网络，作为 IPCC 的附属机构，提供患者护理。 2006 年会议的目标是利用现有的递送技术和药物建立有效的 PC 临床试验所需的步骤和结构。会议将设立一个论坛，讨论 PC 基因治疗的研究进展以及模型系统的开发和使用，包括 PC 鼠标的开发。除了 IPCC 成员的进展报告外，各领域专家的意见也将加强该论坛，其中包括寻求其他罕见疾病临床试验的科学家和临床医生。 FDA/OOPD 也将参加会议，提供有效利用罕见药物指定的专业知识。 会议还将有初级研究人员的参与，并将特别努力让妇女和少数族裔参与讨论小组和演讲。