Multiple system atrophy
- Multisystem atrophy
- Proximal spinal muscular atrophy
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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.
Third NIH Workshop on Gaucher Disease and Parkinsonism, Thursday, April 08, 2010 - Friday, April 09, 2010
Location: NIH- the Cloister, Bethesda, Maryland
Description: Workshop goals were to (1) determine the appropriate human, animal, or cell-based models to better understand this correlation; (2) explore the mechanisms and/or pathways involved; and (3) establish international collaborations to enhance progress in the field and to make recommendations for future study.
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.
Workshop on Glucocerebrosidase and the Synucleinopathies, 2004
Location: NIH Fogarty Center, Bethesda, MD
Description: Several new studies have reported evidence for an association between Gaucher disease, the inherited deficiency of glucocerebrosidase, and the synucleinopathies, clinically diverse neurodegenerative disorders characterized by fibrillar α-synuclein inclusions in neuronal and/or glial cell populations. Recently, several groups have reported findings that show an increased frequency of Gaucher mutations in patients with parkinsonism. This conference brought together basic researchers, pathologists, and clinicians from diverse backgrounds studying synucleinopathies and/or Gaucher disease to explore the etiology and significance of this association. The format included presentations from invited speakers and roundtable discussions. Agenda items included talks on clinical, natural history, animal, pathology, cell biology, protein, and molecular studies as well as discussions of strategies for future research and therapeutic advances. A consensus statement will be drafted for publication. Goals included (1) understanding the contribution of mutant glucocerebrosidase to the development of synucleinopathies, (2) better establishing the frequency of this association in different clinical cohorts and determining the relative risk of developing parkinsonism in Gaucher patients and heterozygotes, and (3) raising awareness of how heterozygosity for a rare disease may be a risk factor for common disorders.