A powerful movement is taking shape in the U.S. rare disease community that could transform the lives of millions of people. That’s right — millions. Even though a single rare disease may affect only a few individuals, there are several thousand of these problematic diseases that are difficult to identify and treat.
Since 2015, 21 U.S. states have passed legislation to create Rare Disease Advisory Councils that provide platforms for patients and family members to communicate with experts, policymakers, and the broader public. It’s critical to seize this hopeful moment because the needs of so many people living with rare diseases go unaddressed.