A DAY FOR RARE DISEASES
NEW YORK
RARE DISEASE
OVER 7,000 RARE DISEASES
ONLY FEWER 400 MEDICINES FOR THESE DISEASES
HELP US GET THE 21ST CENTURY
BILL PASSED IN THE UNITED STATES GOVERNMENT
THE PROJECT
We are having A Day For Rare Diseases to help raise awareness for diseases that most don't have a cure but is effecting 1 out of every 10 people in the United States.
Currently, there are fewer than 400 approved treatments for 7000 rare diseases affecting more than 30 million Americans.
A Day For Rare Diseaseswas established to raise awareness with the public about rare diseases, the challenges encountered by those affected, the importance of research to develop diagnostics and treatments, and the impact of these diseases on patients' lives.
THE HARD TRUTH
Rare diseases collectively affect millions of Americans of all ages. They often are serious and life altering; many are life threatening or fatal. Because each rare disease affects a relatively small population, however, it can be challenging to develop drugs and medical devices to prevent, diagnose, and treat these conditions. We still lack even a basic understanding of the cause or underlying molecular mechanisms of many rare diseases. To help in accelerating rare diseases research and product development, the National Institutes of Health (NIH), with support from the Food and Drug Administration (FDA), asked the Institute of Medicine (IOM) to examine the opportunities for and obstacles in developing drugs and medical devices for treating rare diseases.
A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time.
Characteristics of rare diseases
Over 7000 rare diseases are characterised by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient suffering from the same disease.
Relatively common symptoms can hide underlying rare diseases leading to misdiagnosis and delaying treatment. Quintessentially disabling, the patients quality of life is affected by the lack or loss of autonomy due to the chronic, progressive, degenerative, and frequently life-threatening aspects of the disease.
The fact that there are often no existing effective cures adds to the high level of pain and suffering endured by patients and their families.