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Gerard Vockley, MD, PhD, is the site principal investigator for the Sterol and Isoprenoid Diseases Research Consortium (STAIR), which is uniquely focused on studying a group of diseases bound by common biochemistry, impact on health, and rarity. These include:
STAIR activities are performed by a team of investigators chosen for their clinical research strengths and resources, a long history of collaboration, a diverse geographic distribution to allow maximal access by potential research subjects, their individual motivation to improve the health of patients, and the commitment of their institutions to support the consortium.
STAIR will conduct two major clinical studies over five years – a longitudinal natural history study of NPC and a therapeutic trial to evaluate the efficacy of antioxidant therapy in SLOS – as well as six pilot research studies involving patients with SLS, SLOS, CTX, HIDS, or sitosterolemia. Together with the intramural National Institutes of Health program, the consortium will support a full-scale training program in the field of sterol and isoprenoid diseases and share its resources and data with the NIH Rare Diseases Clinical Research Network.
Participating institutions include Oregon Health and Science University (OHSU), Eunice Kennedy Shriver National Institute of Child Health and Human Development, Children’s Hospital of Pittsburgh of UPMC, Cincinnati Children’s Hospital Medical Center, University of Nebraska Medical Center, and University of Manitoba (Canada). OHSU will be the administrative home of the consortium. The following patient-support organizations will participate in STAIR activities: Smith-Lemli-Opitz/RSH Foundation, Hide and Seek Foundation, Ara Parseghian Medical Research Foundation, Dana’s Angels Research Trust, Foundation for Ichthyosis and Related Skin Types, and United Leukodystrophy Foundation. In summary, STAIR will foster multidisciplinary clinical research, promote training and education, and support projects to explore promising leads in the understanding, diagnostics, and treatment of sterol and isoprenoid diseases.
National Institutes of Health
Gerard Vockley, MD, PhD
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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