The National Fragile X Foundation (NFXF) proposes to recruit faculty for its 13th International Fragile X Conference to be held in Miami, Florida, Juy 25-29, 2012. Specifically, the recruited faculty will be those who are engaged in, or have been engaged in, the process of clinical trials of new drugs for the treatment of fragile X syndrome (FXS) and/or who can contribute unique knowledge, insight and/or experience that will enhance the ongoing recruitment and trial process. This conference is the first to occur since the advent of Phase III clinical trials for drugs designed to address the core symptoms of FXS. Families have many questions about participation in the clinical trial process including questions about the potentials benefits and problems resulting from their participation. They also have questions about the possible long- term benefits and whether or not any of these new drugs constitute a """"""""cure."""""""" The NFXF's conferences foster an environment in which families, clinicians and basic scientists meet, mingle and learn from each other. Keynote addresses and breakout sessions are open to all. The majority of sessions are designated as either """"""""family friendly"""""""" or """"""""clinical research."""""""" (Clinical research sessions are highly attended by families and faculty is advised to make their presentations understandable by average family member.) Basic science presentations are routinely attended by parents and, though there is no requirements to do so, speakers often go out of their way to answer questions from parents and to include simple explanations of their complex work. Members of the NFXF's Scientific &Clinical Advisory Committee and its parent-driven Support &Education Committee will have primary responsibility for review of abstracts and faculty selection. Those committees will be asked to include the applicant's contribution to clinical trials education as one of their review criteria. Selected faculty will then be provided travel and accommodation support. Scheduling of sessions will be structured to maximize their stay and interactions with families as well as their fellow professionals. This will partially be accomplished by the NFXF's creation of interactive forums and Q&A panels. The short-term objectives of this proposal are an increase in the number of speakers engaged, in some form or fashion, in clinical trials and the maximizing of those speakers interactions with families and other professionals. The long-term objective is that the network of families impacted by fragile X syndrome will be better informed about clinical trials, more likely to participate, and more understanding about the near-term and long-term pros and cons of new drug development.

Public Health Relevance

Clinical trials of new drugs that address the core problems of fragile X syndrome are now under way. Families impacted by the syndrome have many questions, comments and concerns about the drugs and the trials. The National Fragile X Foundation will bring families together with clinicians at its 13th International Fragile X Conference and will foster the communication between the two groups as part of its overall effort to advance new and improved treatments.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Conference (R13)
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Pediatrics Subcommittee (CHHD)
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Urv, Tiina K
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National Fragile X Foundation
Walnut Creek
United States
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