WORLD Symposium 2009 (Lysosomal Disease Network's 5th Annual Research Meeting)

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DESCRIPTION (provided by applicant): WORLD Symposium 2009, the 5th annual meeting of the Lysosomal Disease Network (LDN) will be held February 18-20, 2009 in San Diego, CA, USA. This 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. 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 investigators are highly focused 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 lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Proof of the synergistic nature of the WORLD Symposium is the multi-center grant recently submitted to the NIH in response to the Rare Diseases Clinical Research Consortia (RDCRC) RFA. That research program includes 18 unique projects, representing 14 institutions. In addition to clinicians and researchers the WORLD Symposium welcomes patients, parents, caregivers, and all patient advocates who wish to attend. This unique feature allows a small, geographically disperse patient population access to information on the latest scientific advances in lysosomal diseases and gives scientists and clinician's unprecedented access to patients outside the clinic setting thus providing a forum to share information that otherwise does not exist. The WORLD Symposium 2009 program will be organized in six platform sessions, two poster sessions, and three 1-hour research panels. This year's Keynote Address will be delivered by Dr. Elizabeth Neufeld, a pioneer in lysosomal disease research. To help ensure a high-quality scientific content, and fulfill the role of NIH involvement in planning, Gary J. Murray, Ph. D. (Program Director, Neurogenetics, Division of Extramural Research, NINDS) is participating on the Program Committee. For each session, the Program Committee has selected two Invited Speakers and selects the remainder of platform and poster-session program from submitted abstracts. PUBLIC HEALTH RELEVANCE: 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.