WASHINGTON, Feb. 23, 2023 /PRNewswire/ —
WHAT & WHEN:
February 28th marks Rare Disease Day – a day designated to driving awareness and change for the more than 25 million Americans and 300 million people worldwide living and struggling with a rare disease. Subject matter experts from the National Organization of Rare Disorders (NORD), America’s longest-standing rare disease patient advocacy organization are available to weigh-in on this important public health matter.
WHY:
- 1 in 10 Americans are diagnosed with a rare disease – half are children.
- Without knowing, and even without a family history, any one of us could have a genetic defect that becomes a rare disease, defined as any disease that impacts fewer than 200,000 patients.
- There are more than 7,000 known rare diseases of which about 80% are genetic; less than 5% have treatments. Even fewer have cures.
- Diagnosis can take five years or more with medical costs three to five times higher than non-rare diseases.
- Patients with rare disease incur nearly half of all health care costs in the U.S.
WHO:
- Peter L. Saltonstall, President and CEO
– 40 years of progress with the Orphan Drug Act, patient advocacy, policy and advancing care and research
– NORD® Rare Disease Centers of Excellence: the first national network of medical institutions and hospitals dedicated to diagnosing, treating and researching all rare diseases, AND training the next generation of rare disease clinicians and scientists.
- Edward Neilan, MD, PhD, Chief Medical and Scientific Officer
– Building a team science model to address colossal gaps in rare disease patient care and R&D
– How NORD is solving rare disease – breaking down barriers to care and drug development
– Training the next generation of rare disease specialists – including medical geneticists
