Director Loeys-Dietz and Marfan for the Marfan Foundation Stacey Watson shares her first-person experience on the Hill today in Washington, D.C.
Today I attended “Patient Advocacy Driving Innovation for People Living with Rare Diseases: A Congressional Briefing in Recognition of Rare Disease Day” hosted by the National Organization for Rare Disorders (NORD).
I was there both as someone living with Loeys-Dietz syndrome and as the Director of Loeys-Dietz and Marfan at The Marfan Foundation — but most importantly, I was there as part of a community.
Rare disease is often perceived as uncommon or isolated. Yet 30 million Americans are living with a rare disease. Individually, our diagnoses may be rare — collectively, we are not. When advocates, clinicians, researchers, and organizations come together, we become a powerful force for innovation and policy change.
Showing up matters. Presence matters. Collaboration matters.
During Rare Disease Week, we are urging Congress to support legislation that directly impacts the rare disease community:
• Access to Genetic Counselor Services Act (HR 6280 / S. 3670)
• Medical Food and Formulas Access Act (HR 5649 / S. 3304)
• Federal Newborn Screening Act
Fewer than 5% of rare diseases have an FDA-approved treatment. Sustained innovation, access to care, and early diagnosis depend on strong federal policy.
Alone we are rare. Together we are strong.
The Marfan Foundation drives research, education and support – and builds community – to improve outcomes, save lives and empower all people to thrive who are living with Marfan, Loeys-Dietz, Vascular Ehlers-Danlos syndromes, and related genetic aortic and vascular conditions.