DESCRIPTION (provided by applicant): The 10th annual WORLD Symposium will be held February 11-13, 2014 (San Diego, CA, USA). This research meeting is a multidisciplinary forum presenting the latest advances in basic science, translational research, and clinical treatments for lysosomal diseases. The theme of the meeting 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; and 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 may 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 lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Evidence of the synergistic nature of the WORLD Symposium can be seen in the growth of the multi-center consortium "Lysosomal Disease Network" (U54 NS065768, a member of the Rare Diseases Clinical Research Network). 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 for sharing information that otherwise does not exist. The program is organized into six sessions, extending from "basic research" through "translational research" to "clinical research." For each session, the Program Committee will select two invited speakers and will fill the remainder of the program from submitted abstracts (the submission deadline is October 1, 2013). For the third consecutive year, the Symposium will include a didactic component, "Lysosomes 101, Fundamentals of Lysosomal Biology and Disease", on the Monday preceding the research meeting (February 10, 2014). Building on-but not supplanting-the curriculum of the previous years, this educational session will offer attendees the basic concepts of the biochemistry, immunology, molecular biology, and genetics of lysosomal disease. In 2014, Lysosomes 101 will expand from 4 hours to a full day of instruction, providing a common understanding for the cutting-edge research presentations that constitute the Symposium content. In order to foster relationships between senior and young investigators, we will once again sponsor 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.

PUBLIC HEALTH RELEVANCE: Treatments for rare, orphan diseases such as lysosomal disorders 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.