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. ? ? ?
