The National Organization for Rare Disorders (NORD) focused a spotlight on individuals and families who display great courage as they live with rare diseases at its Portraits of Courage Celebration last night.
During the program, Brooke and Brielle Kennedy of Michigan, ages 5 and 6, put a medal around the neck of Representative Fred Upton of Michigan for sponsoring legislation to promote and expand pediatric medical research.
A Portraits of Courage gallery displayed photos and stories of 18 individuals and families representing the spirit of the rare disease community. These included Sam and Alex Bode, two sisters from Connecticut living with Friedreich’s ataxia; Joe Ellenberger of Nebraska, living with paroxysmal nocturnal hemoglobinuria (PNH); and Sam Berns, who died earlier this year and who helped promote worldwide awareness of progeria.
“This was a celebration of the spirit of the community,” said NORD President and CEO Peter L Saltonstall. “We meet extraordinary people every day in our work at NORD, and we chose this way of honoring the courage that our patients and families display in the face of great challenges.”
The celebration took place at the National Building Museum in Washington DC.
The program included remarks by Sean Hepburn Ferrer, son of actress Audrey Hepburn and Mel Ferrer, who shared the story of how a rare disease affected his family. Audrey Hepburn died of a rare cancer known as pseudomyxoma peritonei.
In addition to the Portraits of Courage patients and families, honorees included:
PUBLIC HEALTH LEADERSHIP AWARDS:
- Senator Sherrod Brown (OH)
- Representative Fred Upton (MI)
ABBEY MEYERS LEADERSHIP AWARD:
- MPN Research Foundation
LIFETIME ACHIEVEMENT AWARD:
- John Walsh, co-founder of the Alpha-1 Foundation and AlphaNet
PARTNERS IN PROGRESS AWARDS FOR ORPHAN PRODUCTS APPROVED IN 2013:
- Actelion Pharmaceuticals for Opsumit
- Bayer HealthCare for Adempas
- Genentech Inc. for Gazyva
- Genzyme, a Sanofi Company and Isis Pharmaceuticals for Kynamro
- GlaxoSmithKline Plc for Tafinlar and Mekinist
- Pharmacyclics Inc. for Imbruvica