30th Anniversary Celebration
Thank You for Joining Us!
Heroes of the rare disease community joined future advocates on the stage at the glittering 30th Anniversary Celebration of the National Organization for Rare Disorders (NORD) on May 14th at the Mellon Auditorium in Washington DC.
Before an audience of 500 people, Representative Henry Waxman (CA) hailed Abbey Meyers and other patient advocates for their leadership and vision in promoting federal incentives to encourage the development of treatments for people with rare diseases. Rep. Waxman, the author and chief sponsor of the Orphan Drug Act, was one of the honorees for the event, which was a highlight of NORD’s year-long 30th Anniversary observance.
On an evening that many are describing as “magical”, three young representatives of the next generation of rare disease patient advocates also spoke. Megan Barron, a recent Duke University graduate; 11-year-old Brooke Foster; and 8-year-old Ian Brown talked about the rare diseases of particular concern to them – epidermolysis bullosa, mastocytosis, and Langerhans cell histiocytosis – and thanked the rare disease heroes for the work they have done on behalf of patients and their families.
In addition, by three patients and the parent of a patient -- Kathryn Bundy, Christian Jacobs, Reina Weiner, and MaryBeth Krummenacker – presented awards to senior officials from four companies for innovative products approved by the Food and Drug Administration in 2012.
U.S. Deputy Surgeon General Boris Lushniak, MD, served as guest emcee, assisted by longtime NORD adviser and board member Frank Sasinowski. Entertainment was provided by the Howard University Jazz Ensemble and American Idol finalist Siobhan Magnus.
The 30th Anniversary NORD Honorees are:
FOR LEADERSHIP IN PUBLIC POLICY:
- William V. Corr, who served as Counsel to a Subcommittee chaired by Rep. Waxman during the Orphan Drug Act hearings and currently is Deputy Secretary of Health & Human Services
- Senator Nancy Kassebaum (KS-retired)
- Representative Henry A. Waxman (CT)
FOR VISION ON BEHALF OF PATIENTS
- Stephen C. Groft, PharmD, Director of the NIH Office of Rare Diseases Research
- Marlene Haffner, MD, MPH, Director (retired) of the FDA Office of Orphan Products Development
- Lars-Uno Larsson, founder of Swedish Orphan International AB and early industry advocate for orphan product development
- Abbey S. Meyers, advocate for the Orphan Drug Act and President Emeritus of NORD
- Jess G. Thoene, MD, rare disease medical expert, advocate, and one of NORD’s founders and original medical advisers
FOR INNOVATIVE ORPHAN PRODUCTS APPROVED IN 2012
- Aegerion, Inc., for Juxtapid for homozygous familial hypercvholesterolemia
- NPS Pharmaceuticals, Inc., for Gattex for short bowel syndrome
- Onyx Pharmaceuticals, Inc., for Kyprolis for multiple myeloma
- Sigma-Tau Pharmaceuticals, Inc., for Cystaran eyedrops for cystinosis
Thank you to our Sponsors!