Press Releases

2019 Global Genes RARE Champion of Hope Award Recipients Announced

June 25, 2019

2019 Global Genes® RARE Champion of Hope Award Recipients Announced

Nine Individuals and Organizations from Around the World will be Recognized for Their Positive Impact on the Rare Disease Community

Aliso Viejo, Calif (June 25, 2019) — Global Genes® is pleased to announce the 2019 award recipients to be honored following the  8th Annual RARE Patient Advocacy Summit on Friday, September 20 at the Sheraton San Diego Hotel & Marina in California. The Summit is the world’s largest educational event for rare disease patients and advocates. Offering four specialized learning tracks and led by more than 100 rare disease experts, the event is an unparalleled experience for patients and champions of patients alike to equip themselves with knowledge for actionable strategies to accelerate change. 

Over 100 individuals and organizations worldwide were nominated by their peers for the RARE Champion of Hope award for their notable efforts in rare disease advocacy, collaboration in research, science, medical care and treatment. From the extraordinary list of nominees, the awardees were chosen by an esteemed panel of rare disease experts to be honored at the Champion of Hope Awards dinner on September 20, 2019, following the RARE Patient Advocacy Summit. 

Global Genes congratulates the following 2019 RARE Champion of Hope award recipients: 

  • RARE Champion of Hope – Advocacy: Prasanna Shirol, MBA, Executive Director and Co-founder, Organization for Rare Diseases India
  • RARE Champion of Hope – Advocacy: Kristin Smedley, President and Co-founder, Curing Retinal Blindness Foundation
  • RARE Champion of Hope –  Advocacy: James Valentine, Associate Attorney, Hyman, Phelps & McNamara, P.C.
  • RARE Champion of Hope – Advocacy: Tracy and Jennifer VanHoutan, Co-founders, Noah’s Hope
  • RARE Champion of Hope – Collaboration in Research: International Myositis Assessment and Clinical Studies Group (IMACS)
  • RARE Champion of Hope – Medical Care and Treatment: Martina Bebin, MD, MPA, Professor and Director, University of Alabama, Birmingham Tuberous Sclerosis Clinic
  • RARE Champion of Hope – Research and Treatment: Cystinosis Research Foundation (CRF)
  • RARE Champion of HopeRising Star: Dana Perella, Founder, Cookies4Cures
  • RARE Champion of HopeScience: Charles Eaton, MD, Executive Director, Dupuytren Research Group

For more information and details on the above award recipients please visit:

Global Genes is pleased to welcome Champion Sponsor Horizon Therapeutics, Exhibitor Pavilion Sponsor PRA Health Sciences, Presenting Sponsor Takeda and Title Sponsor Mallinckrodt Pharmaceuticals for the 2019 RARE Patient Advocacy Summit. 

Summit Sponsors also include Platinum Sponsors Genentech, Gilead Sciences, Inc., Illumina, Retrophin, Gold Sponsors Abeona, Akcea Therapeutics, Alnylam Pharmaceuticals, Amicus Therapeutics, Inc., AveXis, BioMarin, BridgeBio, Daiichi-Sankyo, Ipsen, Novelion Therapeutics, Pfizer, Silver Sponsors Agios Other 2019 sponsors include Aldevron, AllianceRX Walgreens Prime, Audentes Therapeutics, Inc., Avrobio, Blueprint Medicines, Catalyst Pharmaceuticals, Epizyme, Intercept, Neurocrine Biosciences, Novartis Pharmaceuticals, PTC Therapeutics, Recordati Rare Diseases Inc., Regeneron Pharmaceuticals, Inc., REGENXBIO, Sangamo, Sobi, Spark Therapeutics, Strongbridge Biopharma, UCB, Ultragenyx Pharmaceutical, and Vertex Pharmaceuticals. Agios, Avexis, Avrobio, Catalyst Pharmaceuticals, Enzyvant, Horizon, Mallinckrodt Pharmaceuticals, Neurocrine, Rare Revolution Magazine, and Retrophin.

To learn more about Global Genes and event sponsorship opportunities please contact Global Genes at [email protected]


About Global Genes® 

Global Genes is a 501(c)(3) nonprofit organization on a mission to connect, empower and inspire the rare disease community. We provide hope for more than 350 million people affected by rare disease around the globe. To date, we’ve educated 6 million people in 100 countries about rare disease, equipped 30,000 patients and advocates with tools and resources, and provided $400,000 in support for innovative patient impact programs. This is just the beginning of what we can achieve together. Visit to get involved today.