- Congenital muscular dystrophy with integrin alpha-7 deficiency
- Congenital muscular dystrophy with ITGA7 deficiency
- Intestinal lipodystrophy
- Intestinal lipophagic granulomatosis
- Secondary Non-tropical Sprue
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ORDR Co-Sponsored Conferences
The Spectrum of Caregiving and Palliative Care in Rare Diseases, Tuesday, June 09, 2015
Location: Building 31, 6C, Room 6, Bethesda, MD
Description: The findings and recommendations resulting from the Workshop will provide guidance to the extramural community as well as to NINR and other Institutes, Offices (e.g., ORDR), and agencies in developing strategies for advancing the science of palliative care and caregiving in rare disease and research programs. It is anticipated that a funding opportunity announcement will result from the Workshop.
American Society of Gene & Cell Therapy (ASGCT) 18th Annual Meeting, Wednesday, May 13, 2015 - Saturday, May 16, 2015
Location: New Orleans, LA
Description: From our plenary lectures, this year to be given by Drs. Steven Rosenberg and Anthony Atala, to our 19 scientific symposia featuring invited speakers from across our discipline, to our nightly abstract poster receptions, where the majority of the over 700 abstracts submitted to the Annual Meeting are presented, the ASGCT Annual Meeting is certain to be the premiere scientific meeting in our field.
RDCRN 3rd Conference on Clinical Research for Rare Diseases, Tuesday, October 02, 2012
Location: Hilton Washington DC/Rockville Hotel & Executive Meeting Center, Rockville, MD
Description: Clinical research in rare diseases presents a number of challenges and unique issues that are not usually considered in the training of clinical investigators through existing training programs. Goals of the conference include: direct instruction of trainees and new investigators in rare disease research methodology; development of a reusable curriculum/syllabus on rare disease research methodology; and stimulation of ideas regarding the unique issues facing investigators engaged in the study of rare diseases.
VI International Conference on Rare Diseases and Orphan Drugs (ICORD 2010): Global Approach to Accessibility in Rare Diseases, Orphan Drugs and Neglected Diseases , Thursday, March 18, 2010 - Saturday, March 20, 2010
Location: Palais Rouge Convention Center, Palermo, Buenos Aires, Argentina
Description: For the first time, the VI International Conference on Rare Diseases and Orphan Drugs (ICORD 2010) was convened in the southern hemisphere in agreement with its aim of globalization of rare diseases research and orphan products development activities.
Adaptive and Innate Immune Responses to Neglected Tropical Diseases, Sunday, January 10, 2010 - Monday, January 11, 2010
Location: San Diego Airport Hilton Hotel, San Diego, CA
Description: The goals of this workshop were to (1) obtain an update on recent developments in the fields of basic immunology, immune responses to neglected tropical disease (NTD)-causing agents, and pathogenic mechanisms of NTDs; (2) determine what are the knowledge gaps and under-emphasized research areas for certain diseases that may not be receiving particular emphasis and how to stimulate research in these areas; and (3) develop a prioritized future research agenda for these areas. Plans are to publish results in the form of a report that could serve as a guide for donors and sponsors to prepare RFAs and for scientists to refocus their research efforts.