National Organization for Rare Disorders, Inc.

Each year, the organization provides seed-money grants to academic scientists for scientific and/or clinical research that will ultimately lead to new diagnostics and treatments for rare diseases, for which there are few other sources of funding. Requests for proposals are made throughout the year, and are disease-specific (as funding becomes available for research on specific diseases and disorders)

Research grants and fellowships to academic scientists and physicians specializing in rare diseases. Support to individuals diagnosed with specific rare diseases, to obtain medication and treatment

NORD works closely with humanitarian-minded pharmaceutical and biotechnology companies to ensure that certain vital medications are available to those individuals whose income is too high to qualify for Medicaid but too low to pay for their prescribed medications. NORD’s programs have set the standards for fairness, equity, and unbiased eligibility. The NORD programs have gained respect from the patient communities, pharmaceutical companies, healthcare professionals, government officials, and the public. Participants have the assurance that NORD protects their confidentiality. There is no conflict of interest with respect to NORD’s eligibility decisions.

NORD has been a trusted source for providing critical educational resources to the rare disease community. NORD’s educational initiatives include innovative partnerships and programs to empower patients and their families, inform students of all ages, and support the vitally important work of physicians and other healthcare professionals. Some of the educational programs and resources we offer for patients, students, and medical professionals include: Rare Disease Database: The Rare Disease Database provides reports on more than 1,200 rare diseases for patients and their families. Continuing Medical Education: This program will raise diagnostic awareness of typical patient symptoms and treatments that are actually atypical and continue to manifest over time. Diagnostic resources, tools and detailed infrastructure of resources for both providers and patients will be demonstrated in this program. Students for Rare: Graduate and medical students can launch a student chapter program at their university to get students informed and interested in rare disease research and awareness. Additionally, high school students and undergraduate medical students have the opportunity to launch a NORD student club at their school to promote rare disease education and awareness. Joint Publications with NORD: Through its partnerships with Frontline Medical Communications and Medscape, NORD provides useful medical publications and video resources for clinicians as well as researchers.

NORD’s Research Grant Program provides seed grants to academic scientists for translational or clinical studies related to the development of potential new diagnostics or treatments for rare disease. In at least two cases, NORD grants have resulted ultimately in FDA-approved treatments for patients. The studies funded with NORD research grants provide preliminary data, on drugs, devices or medical foods that researchers may then use to attract funding from government or industry sponsors to pursue further clinical studies.

Starting with the Orphan Drug Act in 1983, NORD’s mission has always included ensuring that the laws, regulations, and incentives surrounding the drug development process encourage orphan therapy development. For over 35 years, NORD has worked to strengthen the orphan drug development process. NORD works with patients, patient organizations, Congress, the FDA, and the biopharmaceutical industry to strengthen the orphan drug development landscape. NORD’s Rare Action Network® (RAN) is the nation’s rare disease advocacy network working to improve the lives of the 30 million Americans impacted by rare disease. The mission of RAN is to connect and empower a unified network of individuals and organizations with tools, training, and resources to become effective advocates for rare diseases through national and state-based initiatives across the United States.