2013 International Pachyonychia Congenita Consortium (IPCC) Symposium

描述（由申请人提供）：本申请申请为第十届国际先天性厚甲联合会（IPCC）题为“实施基于研究的先天性厚甲（PC）临床研究”的研讨会提供支持。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

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.