From Left to Right: Mary Dunkle- NORD, Christy Greeley- Cystinosis Research Network, Ron Bartek-Friedrich’s Ataxia Research Alliance, Jeff Wuchich- Alternating Hemiplegia of Childhood Foundation, Mirta Avila Santos, American Behcet’s Disease Association (ABDA), Sandy McElgunn- ABDA, Megan O’Boyle- Phelan-McDermid Syndrome Foundation

Within the first hour of the Annual DIA-NORD U.S. Conference on Rare Diseases & Orphan Products, NORD and DIA hosted a panel of six patient advocates to help remind everyone that patients are at the heart of everything we will be talking about in the next few days.

Over the course of the conference, we will hear speakers talk about major themes in research and regulation, policy and challenges in the rare disease space, which can often become very academic. However, this reminder of the human face of the topics will help ensure that participants in this session don’t lose sight of the ultimate purpose of our work, to improve the lives of rare diseases patients and the incredible commitment patient advocates have to drive results.

As Megan O’Boyle, a parent and patient advocate from the Phelan-McDermid Syndrome Association, said:  “I am not a doctor, lawyer, pharmacist, scientist, or drill sergeant; but every day I am each of these things.” Patient advocates play an integral role in advancing knowledge of their disease and driving success in research and orphan drug development.

We are living in a time of accelerated drug approvals, significant policy achievements, and advances in science that researchers are calling the most exciting time in scientific discovery.  However, we know the work still in front of us is vast.  For these patient advocates and the millions of people affected by rare diseases, there is no choice but to move forward.  The lives of our loved ones are at stake.

It is through collaboration and the opportunity to share ideas and best practices at conferences such as this, that we will see advancement and progress.  As Ron Bartek of the Friedreich’s Ataxia Foundation said, “Acting alone, there is precious little we can accomplish; acting together, there is precious little we will not accomplish.”