Goals of the symposium include working together to (1) gather information/data to support new clinical trials with drug/gene therapies based upon in vitro and in vivo research results; (2) generate important reagents, including antibodies, animal models, cell lines, tumor samples, recombinant proteins, etc., for distribution to the BHD research community;...
Events
The goal of this conference is to advance the field of craniofacial morphogenesis and regeneration and treatment of rare craniofacial diseases by communicating the most recent findings from the field. The interdisciplinary group of scientists working in basic science and translational fields will encourage cross-fertilization of ideas, experimental systems, animal...
The goals of this conference are to extend the findings in the area of ATM’s role in the DNA damage response into a better understanding of the neurologic phenotype of A-T. For information and registration please visit the Conference Web Site
February 28, 2010, is RARE DISEASE DAY, a series of events in Basel and Zurich designed to raise awareness among the general public about rare diseases and their impact on people’s lives. Throughout the day there will be special celebrity appearances and prizes given away, as well as a children‘s...
The broad objectives for the 2010 LAM Research Conference are to review research developments in LAM; attract new investigators to LAM research; identify promising new LAM research directions; and attempt to reach consensus on difficult clinical issues. The investigators believe that a 2010 LAM Research Conference is needed to sustain...
Workshop goals are to (1) determine the appropriate human, animal, or cell-based models to better understand this correlation; (2) explore the mechanisms and/or pathways involved; and (3) establish international collaborations to enhance progress in the field and to make recommendations for future study.
Themes of ECRD 2010 1. National strategies and plans for rare diseases 2. European Reference Networks and Centres of Expertise for Rare Diseases 3. Science from the bench to the bed side 4. Information and Medical Education 5. The European Committee for Rare Diseases 6. Policy scenarii for rare...
The goals of this workshop are to (1) assess the state of knowledge regarding the musculoskeletal aspects of aging with OI; (2) explore the potential for new diagnostic and therapeutic techniques for OI; and (3) build a consensus on essential measures for documenting OI treatment outcomes.
For the first time, ICORD — the International Conference on Rare Diseases and Orphan Drugs — will be convened in the southern hemisphere in agreement with its aim of globalization of rare diseases research and orphan products development activities. For information and registration please visit the Conference Web Site.
The goal of this meeting is to mentor the career development of a “hybrid” junior scientist working in developmental disabilities research, including those within T32 training programs as well as other training programs. The focus is on pre- and post-doctoral students (“scholars-in-training”) because interdisciplinary training after scientific formation appears to...
This conference is expected to generate immediate and long-term contributions to clinical care and basic and translational research similar to those generated by previous conferences. Outcomes from previous conferences have dramatically expanded awareness of this disease among physicians and scientists, led to improvements in patient care and new clinical trials,...
The overall objectives of this workshop are to: (1) define NA and NBIA research priorities (2) determine resources that are needed in order to foster research (3) stimulate interest in research into NA and NBIA among scientists in related fields (4) generate collaborations between those working in related disciplines, with...
Advances in ataxia research are occurring at a rapid pace. Given the breadth of pathogenic processes, methods of evaluation, and clinical and basic science approaches that pertain to ataxia research, there is a clear need for a continued forum, namely AIM2010, at which these multifaceted elements of ataxia research can...
Anticipated goals and use of results: Formulation of strategy for advancing clinical research in PAH will be high priority. Recommendations by the participants will be made available via publication in a peer-reviewed journal. Initiatives on future research will be considered.
Specific Aims of this meeting are to: 1.) Emphasize the strategies for, and identify the obstacles to, moving from translational research to clinical trials. 2.) Coalesce members of the LD network into functional research collaborations and to present to the LDN community progress on the specific projects that are part...
The effectiveness of this workshop will be assessed by tracking the number of collaborations established between investigators and incorporation of these investigations into clinical protocols. The number of objectives relating to osteonecrosis in future ALL protocols will be tracked and could impact quality-of-life issues and cancer survivorship. The incidence of...
The goals of the workshop include: 1) Establish a protocol for standardized collection of first and serial neurological and neuropsychological data on FTD patients within the interested ADCs. 2) Establish FTD-specific pathology data elements, and determine whether the ADC pathology protocol addresses these elements, or modification is needed for a...
Registration is now open for the second Bone Marrow Failure Disease Scientific Symposium to be held March 11 – 12, 2010 at the Bethesda Hyatt Hotel in Bethesda, Maryland. The symposium is being presented by the Aplastic Anemia & MDS International Foundation and sponsored by the NIH National Heart, Lung and Blood Institute...
Featuring a stimulating and progressive programme disseminating the most recent research in the field of neuromuscular conditions, this conference will provide an exciting poster session where up and coming researchers will have an opportunity to present their research to the neuromuscular community. for further details
Improving Access to Orphan Drugs for all Patients Affected by Rare Diseases in Europe: EU Assessment of Clinical Added-Value of Orphan Drugs (CAVOD) For more information: article