Aliso Veijo, California — June 26, 2023 — Members of the rare disease community from around the world will gather in San Diego this September for Global Genes’ 12th Annual RARE Advocacy Summit, one of the largest gatherings of the rare disease community. 

The event is an opportunity for advocates to learn, network, and inspire each other as they interact with researchers, healthcare professionals, drug developers and other advocates during the three-day event that runs September 19 through 21 at the Sheraton San Diego Hotel & Marina on Harbor Island Drive in San Diego. The RARE Advocacy Summit (formerly known as the RARE Patient Advocacy Summit) anchors Global Genes’ Week in RARE, a series of events that will take place at the venue including the RARE Health Equity Forum, the RARE Champions of Hope Awards, the Global Advocacy Alliance Meeting, and the RARE Corporate Alliance Meeting. 

Pulitzer Prize-winning health and science reporter for The Wall Street Journal Amy Dockser Marcus will deliver the opening keynote address. Marcus is author of We the Scientists, which tells the story of a group of parents of children diagnosed with the rare and fatal genetic lysosomal storage disorder Niemann-Pick disease type C. When these families were confronted with the fact that no treatment existed or would likely be developed in time to save the lives of their children, they began collaborating with each other, researchers, physicians, and even former NCATS director, Dr. Chris Austin, to accelerate along the path to a treatment. 

“Amy’s book tells the story of the rise of citizen scientists and shows how one group of patient families catalyzed and advanced the development of therapies for a rare and deadly disease,” said Charlene Son Rigby, CEO of Global Genes. “It’s a fitting way to begin the RARE Advocacy Summit, which is about connecting advocates to the knowledge, resources, and partners they need to change what it means to live with a rare disease.”

Susannah Fox, a leading expert in healthcare and technology, will deliver the closing keynote. Fox’s book, Rebel Health: A Field Guide to the Patient-Led Revolution in Medical Care, will be released in February 2024. During her time at Pew Research Center, Fox studied the impact that technology could have on healthcare, including how patients and caregivers were crowdsourcing diagnoses. Her book will further examine how patients and caregivers are leading the charge for peer-to-peer healthcare, and how those in healthcare can join. Fox previously served as Chief Technology Officer for the Department of Health & Human Services during the Obama administration.

This year’s summit will consist of four tracks. They include: 

• Empowering the RARE Individual, which focuses on helping rare disease patients more effectively navigate the world of healthcare; 
• Community and Capacity Building, which will help rare disease organizations expand their reach and improve their effectiveness; 
• Becoming a Research Ready Organization, which will help patient advocacy groups develop and execute a research agenda to accelerate the development of therapies; and 
• Rare Disease Master Class: Science and Tech Innovation, which will provide a series of discussions with drug development experts guiding rare disease advocates through the different therapeutic modalities. 

In addition to the keynotes and the sessions falling within these four tracks, attendees can take advantage of one-on-one time with leaders within the industry during Expert Office Hours, take part in “Birds of a Feather” networking, and Freshman Orientation for first-time attendees.

The Champions of Hope awards ceremony will take place on the evening of September 19, the first day of RARE Advocacy Summit. The three Champions of Hope will be honored for raising the bar through their innovative approaches to research, programming, and advocacy to create a meaningful impact in the rare disease space.

The RARE Advocacy Summit is sponsored by Travere Therapeutics, Genentech, Horizon Therapeutics, Pfizer, Chiesi, Sanofi, Vertex, Alexion, AllianceRx, Amicus Therapeutics, Avidity Biosciences, BioCryst, Biomarin, Janssen, Jazz Pharmaceuticals, Pharming, Regeneron, Spark Therapeutics, BridgeBio, Catalyst Pharmaceuticals, Ovid Therapeutics, Sangamo, Rosamund Stone Zander Translational Neuroscience Center at Boston Children’s, Merck, Ultragenyx, Moderna, Neurocrine Biosciences, and PTC Therapeutics. For more information about the RARE Advocacy Summit, please visit https://globalgenes.org/event/rare-advocacy-summit/. 

About Global Genes
Global Genes is a 501(c)(3) nonprofit rare disease patient advocacy organization dedicated to providing patients and advocates with a continuum of services to accelerate their path from early support and awareness through research readiness. Using a collaborative approach that involves biopharma, researchers and advocates with data as a central core, Global Genes also enables research and data collection through the RARE-X research program. Through this effort, Global Genes is building the largest collaborative patient-driven, open-data access initiative for rare diseases globally.

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