Research

Research Grant Program

NORD's Research Grant Program provides seed money in small grants to academic scientists studying new treatments or diagnostics for rare diseases. The clinical researchers supported by NORD's research grants provide preliminary data indicating that a treatment (drug, device, or medical food) may be safe and effective when used for a larger number of patients. Researchers can then use the preliminary data to apply for larger multi-year government grants or to attract a commercial sponsor who will manufacture an orphan product and get it approved for marketing by the U.S. Food & Drug Administration (FDA).

Rare disease research funding opportunities being made available through NORD.

Rare disease research funding opportunities being made available through NORD Member-Organizations.

Read NORD's Research Program Policy to learn how NORD helps individuals, families, groups, or organizations sponsor research.

NORD Awards Research Grants

NORD's Medical Advisory Committee has recommended, and the NORD Board of Directors has approved, the awarding of seven research grants in November 2008. The patient organizations that funded the grants (when applicable), investigators, their institutions, and their projects are as follows:

Alveolar Capillary Dysplasia

(Alveolar Capillary Dysplasia Association)
Partha Sen, PhD
Baylor College of Medicine
Genotyping and VEGF188 Expression Study for Further Understanding of the Molecular Basis and Pathophysiology of ACD

APS Type 1,(Autoimmune Polyglandular Syndrome, Type 1)

(APS Type1.Org)
Daniela Cihakova, MD, PhD
Johns Hopkins University
Chronic Candidiasis Develops in the Absence of Normal Aire Function Due to a Defect in the Induction of IL-17 by Dendritic Cells

Matthias Wabl, PhD
University of California, San Francisco
Hypermutation as a Cause of Autoimmunity in Aire Deficiency

Olivopontocerebellar Atrophy and Related Disorders

Henry Houlden, MD, PhD
Institute of Neurology, London
The Identification and Characterization of Disease Genes and Risk Factors that Predispose to Multiple System Atrophy (MSA)

Puneet Opal, MD, PhD
Northwestern University
Molecular Pathways Underlying SCA1 Degeneration

Tarlov Cysts

Tarlov Cyst Disease Foundation
Anne Louise Oaklander, MD, PhD
Massachusetts General Hospital/Harvard Medical School
Early Detection of Risk Factors for Tarlov Cysts and Their Complications

Tyrosinemia Type 1

Joshua's Cure.Org/Danielle Barckett Fund
Eugene Swenson, MD, PhD
Yale University School of Medicine
Bone Marrow Mesenchymal Stem Cell Transplantation for Hereditary Tyrosinemia Type 1