June-Rare Diseases
Innovation.org Highlights Research in Rare Diseases
According to the National Institutes of Health Office of Rare Diseases, there are 6,000–7,000 rare diseases affecting a total of 25 million Americans. This population is particularly in need of medicines and other treatments because, as the U.S. Food and Drug Administration estimates, 85-90% of rare diseases are serious or life-threatening. These vital new treatments are often categorized as “orphan” drugs, or products that are used to treat patients with “orphan” or rare diseases (a disease that affects fewer than 200,000 Americans). With the passing of the Orphan Drug Act in 1983, researchers have made great strides in developing effective treatments for people living with rare diseases.
Several national health observances are being held in June for different rare diseases. The U.S. Department of Health and Human Services recognizes National Awareness in June for Myasthenia Gravis, a chronic autoimmune neuromuscular disease that is characterized by different degrees of weakness in the skeletal muscles; Aphasia, a communication disorder that disrupts a person’s language processing skills; and Scleroderma, often called “systemic sclerosis”, a chronic disease of the connective tissue.
Recognizing the impact that rare diseases have on the American population, Innovation.org highlights important and innovative research into rare diseases and treatments. Click on the links below to read more about rare disease research.
Introduction to Rare Diseases
Click here to learn more about rare diseases, including treatments and progress in current research.
Click here to learn about key facts and figures in rare diseases and orphan drugs.
Advances in Rare Disease Research
Patient Perspectives
Today there are many treatments for rare diseases that greatly ease the burden for those living with the disease.
Click here to read thoughts from patients living with rare diseases and to learn how advances in treatments impact patient lives.
Milestones
Between 1995 and 2005, over 160 drugs for rare diseases were approved.
Click here to see a timeline highlighting key milestones in rare disease research and treatments. Just as the number of new orphan drugs being approved has grown, the number currently being studied continues to rise. According to the
FDA's Office of Orphan Products Development, in 2004 there were a record 160 applications for orphan status among drugs in development.
Click here to read about future trends in rare disease research.
Treatments for Specific Rare Diseases
Click on the links below to learn more about innovative research for specific rare diseases.
Featured Report
A Decade of Innovation: Advances in the Treatment of Rare Diseases (April 2006)
New Medicines in Development
PhRMA, with the collaborative support of Genetic Alliance and the National Organization for Rare Disorders (NORD), (
Click here for more information about these two partners) published a report in February 2007 which found that there are more than 300 medicines currently in development to treat or prevent hundreds of rare diseases.
Click here to read the full report and to read information on selected examples of orphan drugs in development for rare diseases.
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