On Friday, May 6, 2016 I had the pleasure of attending and representing Cure AHC at the Global Genes Rare Patient Advocacy Symposium. Global Genes collaborated with the University of Pennsylvania Orphan Disease Center (ODC) to make this half-day event possible. The symposium covered four main topics: patient engagement in drug development, building a plan to pursue research, finding and raising funds for research, and how to grow your organization.
Several guest speakers were on hand to inspire, educate, and motivate. David Fajgenbaum, MD, MBA, MSc, shared his personal experiences with Castleman disease, Kyle Bryant spoke of living with Friedreich’s ataxia, and Emily Kramer told of her rare form of cystic fibrosis. In addition to rare disease speakers, attendees were able to share their experiences, ask questions, and network.
During my time networking, I found two organizations especially beneficial to the mission of curing AHC. BioPontis Alliance collaborates with researchers and organizations to ensure the needs of the organization are being met, they help organizations partner with pharmaceutical companies, help manage research, and assist in finding government funding and support. The Coriell Institute is a biorepository that houses specimens for researchers. Families are able to donate specimens, free of charge for research.
In sum, I left the symposium with empathy for what many other rare disease organizations are going through, inspired that even more rare diseases are making great strides towards research and building relationships with pharmaceutical companies, and proud to be part of an organization like Cure AHC, which is dedicated to providing support to families and research. I would like to encourage you all to spread awareness and to raise money. It can be as simple as sharing your champion’s story in our newsletter or starting a Mobile Cause page to obtain donations online. Please know that I am happy to help anyone find the perfect fundraising fit for your family and champion. All you have to do is reach out to me at firstname.lastname@example.org.