The 4th annual WORLD Symposium will be held February 13-15, 2008 in Las Vegas, NV, USA. In addition to the annual meeting of the Lysosomal Disease Network (LDN), the symposium will provide a multidisciplinary forum to present and discuss clinical research to foster new understanding of the molecular biology, pathology, and potential for treatment for these diseases.
Specific aims are to: 1) Further coalesce members of the LDN as a functional research network, 2) Foster interdisciplinary collaboration with the overall goal of improving knowledge of basic discoveries and clinical manifestations of these diseases; 3) Identify and discuss the latest findings in the natural history of lysosomal diseases (LDs), diagnostic testing and screening, and treatment; and 4) Identify areas requiring additional basic/clinical research, public policy and regulatory attention. Many of the network's constituents investigate and/or treat specific aspects of these diseases but have little exposure to work done in areas outside their current area of interest. This meeting platform promotes the sharing of basic and clinical science advances for all LDs, and provides an opportunity to discuss the related treatment outcome issues. The study and treatment of LDs pose unique challenges due to small populations of patients being dispersed over a wide geographic area. Providing a forum to share information in the basic and clinical sciences will advance research in both realms, and result in a more effective and efficient dissemination of knowledge, clinical guidelines, and recommendations for diagnosis, evaluation, and treatment. The 2008 WORLD Symposium program will be organized in five sessions. Session I: Basic Science, Advances in Understanding the Pathophysiology of Lysosomal Diseases. Session II: Basic Science, Future Therapeutic Approaches to Treatment of Lysosomal Diseases. Session III: Translational Research, Innovative Therapies Moving Toward Clinical Trials for Lysosomal Diseases. Session IV: Clinical Trials, Experimental Therapies Currently Being Tested. Session V: Natural History, Treatment Outcomes, and Quality of Life Issues. Additional concurrent disease-specific sessions on the afternoon of the last day (of special interest to patients, parents, and patient advocates) will include educational presentations, support services, and round table discussions. To help ensure a high-quality scientific content, and fulfill the R13 requirement for NIH involvement, Dr. Danilo Tagle (NINDS) has agreed to participate on the Program Committee. Additional, intramural and administrative NIH personnel are being recruited to supplement LDN Steering Committee members who thus constitute the Program Committee. For each session, the Program Committee will select two Invited Speakers and fill the remainder of the program from submitted abstracts (abstract submission deadline June 1, 2008; program finalized July 1, 2008).
New, expensive treatments are developed for the rare, """"""""orphan"""""""" lysosomal diseases, some life-long treatments costing $200,000 - $1,800,000 per year for each individual. For others, there are no treatments; such slowly progressive physical and neurologic disorders cry out for innovations in therapy. The WORLD Symposium brings together researchers, physicians and patient advocates to advance medical science and public policy for such desperate problems. ? ? ?
