The 6th annual WORLD Symposium will be held February 10-12, 2010 in Miami, FL, USA. This year, for the second straight year, the Symposium is being co-organized by NINDS. This research meeting is a multidisciplinary forum presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases (LD). The theme of the meeting """"""""Transitions from Translation to Trials"""""""" emphasizes the primary aim which is to assess the mechanisms, and obstacles, for taking bench research into human therapy. Additional aims build upon this, and implement the goals of the Lysosomal Disease Network (LDN) to: 1) Further develop the mechanisms and mission of the LDN;2) Foster interdisciplinary collaboration between scientists, leading to improved knowledge regarding the biochemical, immunologic, genetic, and clinical manifestations of these diseases;3) Identify and discuss the latest findings in diagnostic testing, screening, and treatment;4) Identify areas that need more basic/clinical research, public policy, and regulatory attention. Because many LDN constituents investigate and/or treat specific aspects of these diseases they have little exposure to work done in areas outside their current area of interest. This meeting allows for the sharing of knowledge and advances for all LD, and provides an opportunity to discuss treatment outcomes. Proof of the synergistic nature of the WORLD Symposium is the multi-center grant recently funded by the NIH in response to the Rare Diseases Clinical Research Consortia (RDCRC) RFA. The RDCRC includes 18 unique projects, representing 14 institutions. In addition to clinicians and researchers the WORLD Symposium welcomes patients, parents, care-givers, and all patient advocates who wish to attend. This unique feature allows a small, geographically divergent patient population access to information on the latest scientific advances in LD and gives scientists and clinician's unprecedented access to patients outside the clinic setting;providing a forum that otherwise does not exist. The WORLD Symposium 2010 program will be organized in five sessions: Session I and II, Basic Science;Session III and IV, Translational Research;Session V and VI, Clinical Trials. Additionally, each afternoon session will include with a meeting of the Investigators who's projects are a part of the RDCRC. Each day, six P.I.'s will present updates on the progress being made on their particular project. Additionally, these breakout meetings give each investigator a chance to share ideas with the others. This year's Keynote Address will be delivered by a selected by the Program Committee, a pioneer in LD research. For each session, the Program Committee has selected two Invited Speakers and will fill the remainder of the program from submitted abstracts (abstract submission deadline October 01, 2009).
Treatments for rare, orphan diseases such as lysosomal diseases are relatively new and very expensive. Some life-long treatments can cost $200,000 - $1,800,000 per year for each individual. For others, there are no treatments;for such slowly progressive physical and neurologic disorders innovations in therapy are essential. The WORLD Symposium brings together researchers, physicians, patients and patient advocates to advance medical science and public policy for such devastating diseases.
