The 8th annual WORLD Symposium will be held February 8-10, 2012 (San Diego, CA, USA). This research meeting is a multidisciplinary forum presenting the latest advances in basic science, translational research, and clinical trials for lysosomal diseases. The organizing theme of the meeting will emphasize 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 across all lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Evidence of the synergistic nature of the WORLD Symposium has been outgrowth of the multi-center consortium "Lysosomal Disease Network" (U54 NS065768, i.e., a member of the Rare Diseases Clinical Research Network, RDCRN). In addition to researchers and clinicians, the WORLD Symposium welcomes patients, parents, caregivers, 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 lysosomal diseases and gives scientists and clinician's unprecedented access to patients outside the clinic setting;providing a forum to share information that otherwise does not exist. The program is organized in six sessions extending from "basic research" through "translational research" to "clinical research". For each, the Program Committee will select two Invited Speakers and will fill the remainder of the program from submitted abstracts (submission deadline October 1, 2011). For the first time, the Symposium will add a didactic component "Lysosomes 101, Fundamentals of Lysosomal Biology and Disease" on the Tuesday (February 7) preceding the research meeting. This 4-hour educational session offers all attendees the basic concepts of biochemistry, immunology, molecular biology, and genetics of lysosomal disease thus providing a common understanding for subsequent cutting edge research presentations. Also, Lysosomes 101 was designed, in part, to foster interest in lysosomal biology and growth the field of metabolic research among new/young investigators. To further that goal, this year we will be sponsoring travel for young investigators facilitating their attendance of "Lysosomes 101" and the WORLD conference;the mentoring of new scientists is a major goal of the LDN and the RDCRN.
Relevance: Treatments for rare, orphan diseases such as lysosomal diseases are relatively new and very expensive. When therapies are available they are life-long and 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.