What is a rare disease?
A disease is considered “rare” if it affects fewer than 200,000 individuals in the US. The FDA states that over 7,000 rare diseases affect more than 30 million people in the U.S. Many rare conditions are life-threatening and most do not have treatments. Uncovering the genetic roots of rare diseases and using those insights to develop new treatments is an ongoing process. Unfortunately, a long and complex journey to diagnosis is not unique. Living with a rare disease impacts patients and their families and caregivers both physically and mentally.
These activities focus on the diagnostic odyssey, clinical manifestations and emerging therapies of rare diseases. New for 2022, following each Diagnostic Odyssey for Individuals With Rare Diseases webcast, we are releasing an accompanying podcast sharing the personal experience from the patient, family or caregiver perspective. These accredited podcasts provide an in-depth understanding of the challenges and barriers that patients and their caregivers face as they navigate the complexities of the health care system. We invite you to learn more and please give them a listen!