AMWA was honored to participate in the celebration of the 40th Anniversary of the National Organization for Rare Disorders (NORD), the 40th Anniversary of the Orphan Disease Act, and the 2023 NORD Rare Impact Award recipients.
Held on May 4 at the National Portrait Gallery in Washington, DC, this signature event brought stakeholders within the rare disease community together for a memorable evening with inspiring speeches and uplifting music — wonderfully moderated by Emmy Award-winning journalist Peter Alexander. His charge to the attendees was this: “reimagine what is possible for rare diseases.”
Alexander noted the “fierce determination of the rare disease community,” which was palpable across sectors – whether through testimonials about patient and caregiver experiences, advocacy efforts, or therapeutic discoveries. Most apparent was the impact of synergistic partnerships between patients, advocates, practitioners, and industry.
President and CEO Peter Saltonstall reflected on the founding of NORD in the 1980’s, a time before the internet, email, and social media. He credited leaders like Abbey Meyers, lauded as the “mother” of the NORD movement and a housewife turned advocate who became the founding president of NORD. Like many leaders in the rare disease community, Meyers was a parent on a crusade for her child, whose mission would expand to serve the larger community.
Over the recent months, I have learned that the rare disease community is full of parent champions like Abbey, for example 2023 NORD honoree Connie Lee (founder of the Alliance to Cure Cavernous Malformation), Bo Bigelow (co-founder of the Rare Disease Film Festival), and Donna Appell (Executive Director and Founder of the Hermansky-Pudlak Syndrome Network, featured in the film Rare, which was screened by AMWA on Rare Disease Day), to name a few.
Singer-songwriter CeeCee Castro delivered an empowering rendition of “A Hero Comes Along,” celebrating the strength and fortitude of this community whose motto is, “Alone, we are rare. Together, we are strong.”
I have a personal interest in this community through the work of my aunt, Dr. Vivian E. Shih, an internationally renowned researcher in the field of inborn errors of metabolism, author of Laboratory Techniques for the Detection of Hereditary Metabolic Disorders, and past president of the Society for Inherited Metabolic Disorders. Decades ago as a medical student, I knew very little about her field, except for brief references in lectures. When we established a graduate medical education fund in her name and heard from family, patients, and colleagues, I began to appreciate the breadth and impact of her work.
AMWA’s new campaign to raise awareness of rare diseases through film combines our interest in the medical humanities, medical education, and sex/gender specific medicine. While trainees might never encounter a patient with a particular rare disease, films can bridge this gap by bringing patient stories to life in a memorable way. Education through film may mitigate the delayed diagnoses that can be all too common with these conditions. Film can also be a means to better understand the full spectrum of healthcare needs for patients, including the impact on families and caregivers.
To learn more about this initiative or to get involved or partner with AMWA, please visit this webpage.