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Diseases

Genetic and Rare Diseases Information Center (GARD)

Wolman disease


Other Names for this Disease
  • Familial Xanthomatosis
  • Liposomal Acid Lipase Deficiency, Wolman Type
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

News & Events


News


ORDR Co-Sponsored Conferences

  • 2016 Rare Disease Day at NIH, Monday, February 29, 2016
    Location: Building 10, Masur Auditorium, National Institutes of Health, Bethesda, MD
    Description: The 2016 Rare Disease Day at NIH will take place on February 29. The event, sponsored by NCATS and the NIH Clinical Center, aims to raise awareness about rare diseases, the patients they affect and the research collaborations that are addressing rare disease challenges. The day will feature tours, posters and exhibits, and presentations.

  • Gordon Research Conference and Gordon Research Seminar on Lysosomes and Endocytosis, Sunday, June 15, 2014 - Friday, June 20, 2014
    Location: Proctor Academy, Andover, NH
    Description: <p>The main goal of the Lysosomes and Endocytosis GRC is to foster the dissemination of current research results and the establishment of new research areas and new collaborations in the area of the cell biology of endocytosis, lysosomes, endosomes and related organelles. We hope that many of these new directions and collaborations will be directed toward the etiology, diagnosis and treatment of rare genetic diseases such as lysosomal storage disorders, Hermansky-Pudlak syndrome, Chediak-Higashi syndrome, Niemann Pick disease and tuberous sclerosis, among others.</p>

  • 2011 Lysosomal Disease Gordon Research Conference, Sunday, January 23, 2011 - Friday, January 28, 2011
    Location: Hotel Galvez, Galveston, TX
    Description: This Gordon Research Conference had sessions on topics ranging from the basic science of lysosomal biology and function (but with strong emphasis on pathogenic mechanisms of lysosomal disease), relationships of lysosomal diseases to other neurological diseases, pathogenic cascades, biomarkers, recent advances in therapy, and clinical trials and design. In addition, there was a hot topics session (with speakers chosen close to the time of the meeting so as to include the most recent developments) and a poster session (nine short talks chosen at the meeting on the basis of the posters that generated the most interest during the afternoon poster sessions). In addition, a number of short talks were chosen prior to the meeting based on abstract submissions.

  • WORLD Symposium 2010 (Lysosomal Disease Network's 6th Annual Research Meeting), Wednesday, February 10, 2010 - Friday, February 12, 2010
    Location: Miami Hilton Downtown, Miami, Florida
    Description: The specific aims of this meeting were to (1) emphasize the strategies for, and identify the obstacles to, moving from translational research to clinical trials; (2) coalesce members of the LD network into functional research collaborations and present to the LDN community progress on the specific projects that are part of the funded U54 RDCRN grant; (3) foster interdisciplinary collaboration with the overall goal of improving knowledge of basic discoveries and clinical manifestations of these diseases; (4) provide an educational forum for young investigators, clinicians, and researchers in the field; (5) identify and discuss the latest findings in the natural history of lysosomal diseases, diagnostic testing and screening, and treatment, with specific focus on (a) inflammatory components of lysosomal diseases and autophagy, especially in the central nervous system, (b) new treatments of the central nervous systems, and (c) ethics and efficacy in treating the presymptomatic or asymptomatic patient; and (6) identify areas requiring additional basic and clinical research and public policy and regulatory attention, such as ethics and economics, and factors that impact implementation of therapy, including newborn screening.

Other Names for this Disease
  • Familial Xanthomatosis
  • Liposomal Acid Lipase Deficiency, Wolman Type
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.