Autosomal dominant neuronal ceroid lipofuscinosis 4B
- Kuf's disease, autosomal dominant
- Ceroid lipofuscinosis, neuronal, Parry type
- Ceroid lipofuscinosis, neuronal, 4B, autosomal dominant
- Neuronal ceroid lipofuscinosis 4B
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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.
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>
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.