Following this 2-day summit, a subset of attendees will provide recommendations for GenTAC for future directions. In addition, the National Marfan Foundation will sponsor a meeting that will focus on the use of information discussed at the meeting to derive a plan for the future development of therapeutic strategies for...
Events
The goals of this meeting are to (1) develop a consensus set of diagnostic criteria for PFAPA, (2) deepen our understanding of the immunobiology of PFAPA, (3) possibly organize a consortium for GWAS in PFAPA, and (4) develop a multicenter treatment protocol for PFAPA. We expect that the meeting will...
The SMS Research Roundtable Symposia will be held in conjunction with the Sixth International Family Conference on Smith-Magenis Syndrome, “Building Bridges of Hope,” on September 17-20, 2009. The Research Roundtable commences the conference and fosters the partnership between parents and researchers, which directly supports the PRISMS’ mission. The Research Roundtable...
This meeting will bring together individuals involved with conveying complex information to different types of audiences. We hope to engage the audience in discussing issues around defining information quality, choosing content appropriately, and utilizing new technologies effectively for education. The audience will include individuals interested in education, evidence-based care, communication,...
Sickle Cell Disease is an inherited condition characterized by severe bone pains, anemia, recurrent infections and complications of the major organs of the body. This condition is inherited and can be prevented if individuals and the community as a whole take testing seriously. Testing for sickle cell disease carrier state...
EMERGING COMMERCIALIZATION STRATEGIES FOR ADDRESSING THE NEEDS OF ULTRA ORPHAN PATIENTS Accelerate Product Launch and Profitability by Building Regulatory Requirements into Distribution http://www.centrichealthresources.com/2009Conference/ The recent Risk Evaluation and Mitigation Strategies (REMS) requirements implemented by the Food and Drug Administration Amendments Act (FDAAA) have made the drug approval process more challenging...
Understand the Latest Regulatory Policy, Clinical Strategies, Patient Outreach and Commercialization Dynamics Impacting Orphan Drug Development. This annual summit brings together industry stakeholders and key thought leaders in the rare disease and orphan drug development arena. Hear from the FDA on results from the “Science of Small Clinical Trials” eight...
The goal of this meeting is to allow interested individuals to express their opinions about the directions clinical research in thalassemia should take, given past experiences and limitations imposed by NIH budgets and regulatory requirements. These proceedings will also serve as the basis of a Request for Applications (RFA) for...
The objectives of this symposium are to (1) describe the latest findings regarding mitochondrial diseases; (2) share research findings and award new research grants; (3) explore the clinical manifestations of mitochondrial disorders, including diagnosis and treatment considerations, and understand the concepts of mitochondrial disease therapy; and (4) evaluate the links...
The first objective of this conference is to facilitate continued discussion and team building of pediatric hematopoietic cell transplant physicians and pediatric immunologists to work together to develop a North American consortium for treatment of children with PID. The workshop of May 2008 set in motion an important collaborative effort....
The primary focus will be on validated endpoints that are ready for incorporation into clinical trials now or in the near future and on methods to address confounding issues unique to studies of acute GVHD. These will be used by the NHLBI-sponsored Blood and Marrow Transplant Clinical Trials Network (BMT...
The goals of this workshop are to refine the meta-analysis done by the IEDB team and to identify knowledge gaps and further research opportunities. Prior meta-analyses meetings have successfully accomplished both. Epitope information will be examined to identify protective immune responses as well as those that may contribute to disease...
The intent of the meeting is to advance the overall field of cardiovascular development and congenital heart disease through the sharing of information and the facilitation of collaborative investigations. An additional goal is to aid the career development of students, postdoctoral fellows, and junior faculty members by providing a forum...
The CIS Primary Immune Deficiency Consortium Conference will provide new investigators in this field the continued opportunity to enhance career development by learning from leaders in the field of primary immunodeficiency and other young investigators from institutions across the Americas and Europe.
Achieving Successful Delivery of Nucleic Acids to Skin May 5-6, 2009, Montreal, Canada (prior to SID annual meeting) Achieving Successful Delivery of Nucleic Acids to Skin is the focus of the 2009 IPCC conference. Both IPCC members and a select group of experts actively involved in delivery to skin will...
This workshop will be unique in that its primary purpose is to facilitate coordination of translational and clinical aspects of Niemann-Pick disease, type C (NPC) on an international basis. Thus, the goals of this meeting are twofold: first, to promote interactions among basic scientists involved in preclinical testing of potential...
This Summit will focus on a critical and perplexing aspect of LAM: the mechanism of lung destruction in the disease. We will again bring together leaders in relevant fields and leaders in LAM research to educate one another about potential mechanisms of lung destruction in LAM and to brainstorm critical...
The intent of the meeting is to advance the overall field of cardiovascular development and congenital heart disease through the sharing of information and the facilitation of collaborative investigations. An additional goal is to aid the career development of students, postdoctoral fellows, and junior faculty members by providing a forum...
This is the largest cytokine conference in the world. Its goal is to bring together clinicians and basic scientists from academic and industrial laboratories with the purpose of understanding and treating diseases in which cytokines play a role.