The Progeria Research Foundation(PRF) will establish an International Hutchinson-Gilford Progeria Syndrome (HGPS) Medical and Research Database for the purpose of obtaining long-term detailed clinical epidemiological and laboratory data on children with HGPS. HGPS is a rare and uniformly fatal """"""""premature aging syndrome"""""""" in which children die of atherosclerotic heart disease at an average age of 13 years. Presently there is little clinical information available on children with HGPS. PRF is the only organization solely dedicated to medical research on HGPS. PRF is collaborating with the Brown University Center for Gerontology and Health Care Research, a well-established data and research center, in designing the database and the data collection protocol. With input and review by an expert scientific advisory committee, the Center's clinical nurse specialists, who have expertise in medical record abstraction, will implement this protocol for the records obtained in the project. The Engelhorn Foundation for Rare Diseases (EFFORD), a non-profit organization, will serve as the database repository. Data analysis will commence when records have been abstracted and data have been coded for 10 children. Results of analyses and other feedback will be provided to families, physicians and researchers through the PRF and EFFORD websites, information sheets, and publications. The proposed database will assist health care professionals in providing appropriate care for children with HGPS and will provide a better understanding of the variations in phenotype of HGPS. This project will make it possible to compile comprehensive in-depth data on the children with this rare disorder, which will serve as a valuable tool in understanding the basis of this disease, and in turn may lead to improvements in its diagnosis and treatment. It will provide a resource for new research in HGPS and in the aging diseases associated with HGPS such as atherosclerosis.

Agency
National Institute of Health (NIH)
Institute
National Institute on Aging (NIA)
Type
Exploratory/Developmental Grants (R21)
Project #
5R21AG021902-02
Application #
6752067
Study Section
National Institute on Aging Initial Review Group (NIA)
Program Officer
Sierra, Felipe
Project Start
2003-06-01
Project End
2006-02-28
Budget Start
2004-06-01
Budget End
2006-02-28
Support Year
2
Fiscal Year
2004
Total Cost
$100,000
Indirect Cost
Name
Progeria Research Foundation, Inc.
Department
Type
DUNS #
092969927
City
Peabody
State
MA
Country
United States
Zip Code
01961
Gerhard-Herman, Marie; Smoot, Leslie B; Wake, Nicole et al. (2012) Mechanisms of premature vascular aging in children with Hutchinson-Gilford progeria syndrome. Hypertension 59:92-7
Olive, Michelle; Harten, Ingrid; Mitchell, Richard et al. (2010) Cardiovascular pathology in Hutchinson-Gilford progeria: correlation with the vascular pathology of aging. Arterioscler Thromb Vasc Biol 30:2301-9
Gordon, Leslie B; McCarten, Kathleen M; Giobbie-Hurder, Anita et al. (2007) Disease progression in Hutchinson-Gilford progeria syndrome: impact on growth and development. Pediatrics 120:824-33
Gordon, Leslie B; Harten, Ingrid A; Patti, Mary Elizabeth et al. (2005) Reduced adiponectin and HDL cholesterol without elevated C-reactive protein: clues to the biology of premature atherosclerosis in Hutchinson-Gilford Progeria Syndrome. J Pediatr 146:336-41