In 2020, the Pennsylvania Rare Disease Advisory Council initiated a groundbreaking survey which yielded a unique lens into the lives of over 1,200 respondents representing 660 unique rare diseases. This survey goes beyond statistics; it unravels diagnostic journeys, illuminates transportation challenges, and spotlights the profound impact on lives.

Our commitment to transparency, data integrity, and collaboration shines through. The survey paints a vivid picture of the challenges faced by the rare disease community in Pennsylvania.

The survey findings unveiled critical delays in diagnosis, mobility challenges, and the economic strain faced by individuals and families grappling with rare diseases.

The Pennsylvania Rare Disease Advisory Council, working closely with legislative Rare Disease Caucus Co-Chairs, the General Assembly, and key stakeholders, aims to leverage insights from the survey. Together, we can implement targeted initiatives, raise public awareness, and advocate for policies that transform lives.