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Diseases

Genetic and Rare Diseases Information Center (GARD)

Gaucher ichthyosis restrictive dermopathy


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News & Events


NCATS Co-Sponsored Conferences

  • American Society of Gene & Cell Therapy (ASGCT) 19th Annual Meeting, Wednesday, May 04, 2016 - Saturday, May 07, 2016
    Location: Washington, DC
    Description: Save the date and plan to head to Washington, DC on May 4-7, 2016, for the premiere gene and cell therapy conference in the world. Taking place at the Marriott Wardman Park Hotel, the ASGCT 19th Annual Meeting will feature ground breaking clinical trial results, cutting edge technology advancements, social networking events, and much more. Join over 1,900 of the worlds top gene and cell therapy professionals for four full days of educational offerings including plenary lectures given by Dr. David R. Liu and 2015 Japan Prize recipients, Dr. Alain Fischer, and Dr. Theodore Friedmann.

  • Pediatric Dermatology Research Alliance (PeDRA) Annual Conference, Friday, October 18, 2013 - Sunday, October 20, 2013
    Location: Westin O’Hare Hotel , Rosemont, IL
    Description: The goals of the PEDRA annual meetings are to provide an educational platform that will enhance the research skills of PeDRA investigators and establish a forum for the critical review of research proposals developed by PeDRA disease-focused groups and to create a bridge between basic scientists and clinicians to enhance opportunities for research. Each group will identify and prioritize the clinical needs and therapeutic opportunities.

  • 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.

See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.