Who Are We?
Knowledge is Power. The Putting Rare Diseases Patients First!® organization is a 501c3 non-profit, and a registered charity. It has tax exempt status with the IRS and in California, USA. Patients with rare diseases need effective diagnostics and treatments. To bring this about, they must be an active part of the process of developing new diagnostics and treatments. The organization seeks to serve the global rare diseases patient community by providing empowering information to rare disease patients, and the parents of children with rare diseases, about the clinical trial and drug development process.
There are 350 million patients with a rare disease globally. In the US alone there are 30 million people with a rare diseases. On the continent of Europe, there are 30 million people with a rare disease.
Many of these patients suffer for many years, and some even die without a conclusive diagnosis. Even after they receive a diagnosis, many patients with rare diseases do not have access to a treatment that is effective. For most of them, the search for effective new treatments continues despite the growing interest in rare diseases.