1) Disseminate current knowledge of the standard treatment of hairy cell leukemia (HCL), 2) Identify the evidence (or lack thereof) with which current treatment practices are based, 3) determine needs and opportunities for clinical research to improve HCL treatment, 4) disseminate results of newer diagnostic techniques, including flow cytometry, PCR...
Events
The goals of this workshop are to (1) obtain an update on recent developments in the fields of basic immunology, immune responses to neglected tropical disease (NTD)-causing agents, and pathogenic mechanisms of NTDs; (2) determine what are the knowledge gaps and under-emphasized research areas for certain diseases that may not...
The goals of this conference are to (1) facilitate international discussion and team-building of hematopoietic cell transplant physicians and autoimmune disease experts to work together for treatment of patients with autoimmune diseases, (2) focus on the aspects of HCT therapy that are particularly challenging for autoimmune diseases, (3) identify questions...
The goal of this workshop is to help foster communication and create conditions whereby rationale drug and biologic development programs in the neuromuscular diseases can be more effective. The organizers anticipate a peer-reviewed publication that focuses upon conveying the broader, derivative lessons from the conference.
This workshop will provide an important forum for bringing together NIH researchers from across the Institutes and Centers with representatives from the Food and Drug Administration (FDA) as well as professional societies and patient advocates to explore the potential utility of adaptive designs. Additional Information: Conference Web Site
The overarching goal of the conference will be to provide the NHLBI with specific recommendations for future laboratory and clinical research focuses, for clinical trials needed to advance the standard of care, and for the best mechanisms to continue the work of the Bone Marrow Failure Disease Consortium, a successful...
Workshop participants will identify gaps in knowledge, prioritize basic and clinical research questions, identify existing resources that can be applied to Prader-Willi syndrome (PWS) research, identify needed resources and technologies, establish cross-disciplinary and multidisciplinary collaboration, formulate a framework for advancing PWS research, and provide a published report of a comprehensive...
This new meeting (International Conference on Sarcoma Biology) will interface with the Milan translational sarcoma meeting, in that the Milan meeting organizers will provide feedback on the proposed programs, and they will participate (as attendees) in the sarcoma biology meeting. The aim of the International Conference on Sarcoma Biology is...
Several of the symposia and/or individual presentations within the symposia are expected to be published in the refereed literature, especially in Environmental Molecular Mutagenesis or Mutation Research. In additions, the interactions among the numerous highly experienced researchers who will attend this meeting are expected to generate collaborations that will substantially...
The goals of the Working Group are to identify research priorities for translational II research in sickle cell disease. The Working Group is designed to foster cross-disciplinary communication and to ensure that other disciplines and skill sets are included in sickle cell research. These priorities—the study designs, methods, disciplines, and...
The conference in 2009 will be the first opportunity to present and discuss results from the initial TSC clinical trials and additional data from basic research and to discuss plans for new clinical trials utilizing mTOR inhibitors and other drug targets. There are significant research opportunities to be discussed and...
Conference goals are to (a) provide a better awareness and harmonization of the diagnostic criteria of the disease and cofactors that contribute to the evolution of clinical disease associated with the defined genetic defects of autoimmune lymphoproliferative syndrome (ALPS) in the hematology, oncology, immunology, and infectious disease community; (b) establish...
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...
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...