02/28/2026
Despite a blizzard sweeping through the Northeast, nothing kept advocates and lawmakers from Tuesday’s Congressional briefing.
In honor of , NORD convened a standing-room-only session led by CEO Pam Gavin to spotlight the urgent challenges and real solutions shaping rare disease research, diagnosis, and access to care.
The discussion ran more than 30 minutes over as attendees leaned into critical issues including genetic testing, newborn screening, and accelerating treatment pathways for ultra-small patient populations.
Pam was joined by Rohini Kosoglu, Advocacy Chair of The DAND Alliance, who spoke as a rare parent and former government leader, and Theresa Strong, Executive Director of The Foundation for Prader-Willi Research. Theresa shared her journey as both a rare parent and researcher whose natural history study helped support a recent FDA product approval. Their stories were powerful reminders of how advocacy and science together drive progress.
Dr. Ada Hamosh, NORD recipient, renowned geneticist, and Director of the NORD Rare Disease Center of Excellence at Johns Hopkins Medicine, brought the realities of rare disease care directly to Congress.
Through compelling patient stories from her clinic, she underscored what access to coverage for medications and screenings truly means for families. She closed with a clear call to action, outlining the legislation Congress must advance to ensure timely diagnosis, equitable access to care, and sustained innovation for millions of Americans living with rare diseases.
We were proud to have Dr. Hamosh represent NORD’s collaborative network of 46 Rare Disease Centers of Excellence before Congress.
Thank you to everyone who joined us on Capitol Hill for this important briefing. NORD remains committed to working alongside lawmakers on both sides of the aisle to advance meaningful solutions for individuals and families living with rare diseases.
You can take action on federal and state policy alerts today, here: https://bit.ly/3ST0XKV (scroll to the bottom for federal alerts)
Alone we are rare. Together we are strong.
