Neuronal ceroid lipofuscinosis 7
- CLN7 disease
- CLN7 disease, late infantile
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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.
14th International NCL Congress: Supporting US Based Scientists, Wednesday, October 22, 2014 - Sunday, October 26, 2014
Location: Córdoba, Argentina
Description: <p>The specific objectives are to: provide a forum to present the latest NCL scientific, clinical and translational research advances; promote opportunities for student and junior investigators to present their research; maximize the opportunities for interactions between members of the NCL research community through promoting discussion at poster sessions and a lively social program; promote links between NCL researchers and those working in other more common disease that may share similar mechanisms; bring together NCL researchers and clinicians with the NCL parent organizations and affected families, promoting clear and effective communication between these groups; and promote up-to-date diagnoses of NCLs in low and middle income countries.</p>
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.
Gene Transfer and Rare Diseases Workshop, Thursday, September 13, 2012
Location: Rockville Hilton, Rockville, Maryland
Description: The NIH Office of Biotechnology Activities and Office of Rare Disease Research, NCATS, are co-sponsoring a workshop to discuss the advances in and challenges of gene transfer for rare diseases. There have been recent promising clinical successes in gene transfer protocols for rare diseases, such as retinal degeneration, hemophilia, and immune disorders. Investigators will discuss their results to date and opportunities and challenges for extending these successes to other similar rare diseases. The panel discussions will focus on strategies for maximizing opportunities for data sharing or platform development.
13th International NCL Congress, Wednesday, March 28, 2012 - Saturday, March 31, 2012
Location: London, England
Description: The conference will focus on therapeutic approaches for NCLs for which the genes and mutations have been identified and how it relates to intracellular protein trafficking, neuropathology, critical analysis and use of animal and cellular models and how to optimize these resources for therapeutic strategies, use of proteomics and system biology in understanding normal function of NCL genes, and avenues for molecular therapy, particularly on drug, gene and stem-cell based therapies.
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.