Lysosomal diseases represent a group of more than 50 disorders caused by inherited defects in a wide spectrum of proteins. These disorders affect many organ systems, most notably brain, leading to chronic illness and death of affected individuals. Work groups focused on these disorders play a crucial role in stimulating discussion and interactions among experts in the field in order to improve knowledge about the pathophysiology of lysosomal storage diseases to identify novel targets and develop innovative treatment approaches. The Lysosomal Disease GRC has played a crucial role in stimulating this field. Held alternatively at the Hotel Galvez in Galveston, Texas, and at the Renaissance Tuscany Il Ciocco in Lucca (Barga), Italy, we are one of the newest of over 170 annual conferences organized by the GRC, an organization known world-wide for its high-quality, cutting-edge academic conferences. The 2017 Lysosomal Disease GRC will again offer a critical venue for addressing major topics in lysosomal biology, disease mechanisms, diagnosis and therapy, including (i) lysosomal positioning and movement, which is emerging as a critical determinant of many lysosomal functions; (ii) lysosomal membrane proteins that contribute to cell dysfunction across many disorders and pathogenic cascades; (iii) mechanisms of lysosomal storage disorders (LSDs); (iv) lysosomal dysfunction in diseases other than LSDs; (v) new, innovative therapies for correction of lysosomal disease; (vi) diagnostics ad biomarkers for LSDs; and (vii) ongoing and upcoming clinical trials focused on lysosomal disease. As conceived, this conference is unique in the academic calendar and an outstanding complement to other existing lysosomal disease forums in USA and Europe. To address these important topics we have invited, as speakers and discussants, 40 leading and internationally known scientists and clinicians working in the field of lysosomal disease. Each has accepted our invitation and will join what we anticipate to be an additional 160 attendees, fully one-third of whom we anticipate will be junior investigators, postdoctoral fellows and graduate students who are at the early stages of their careers. Our 2017 meeting has again been selected to host a GRS, a special meeting organized and run by graduate students and early postdoctoral fellows. The presence of the GRS insures that the very best and the brightest of the next generation of lysosomal disease researchers will be part of the GRC as well. The discussion and cross fertilization of ideas and approaches occurring as part of the GRC and GRS meetings we believe will accelerate not only our understanding of the role of the lysosomal system in health and in disease, but also move us closer to effective therapies for many of these disorders.
Lysosomal diseases, while individually rare, together exhibit an incidence of 1:5000 to 7000 live births, making them as a group one of the more common families of genetic disorders. Most of the 50 known lysosomal diseases affect the brain, causing years of disability, and are fatal. Few treatments exist, particularly for brain disease. There is an urgent need to better understand these disorders, their links to other neurodegenerative diseases, and to develop new and effective therapies.
