Global Genes and Boston Children’s Hospital Partner for 2025 RARE Drug Development Symposium – Equipping Rare Disease Advocates in Research and Drug Development

This Event Will Build Knowledge, Strategy, and Confidence for Patient Advocacy Leaders to Drive Therapeutic Innovation in Partnership with Life Science Partners and Researchers

WASHINGTON, June 24, 2025 /PRNewswire/ — Global Genes, in partnership with Boston Children’s Hospital, the Rosamund Stone Zander Translational Neuroscience Center (RSZ TNC), and the Termeer Institute, announced the partnership for the 2025 RARE Drug Development Symposium (RDDS), designed to empower rare disease advocates to lead the way in research and therapeutic innovation like never before.

Held in Boston, this 1.5-day event will empower the rare disease ecosystem of advocates, researchers, and life science partners for a hands-on, collaborative learning experience focused on accelerating progress in rare disease drug development. With an emphasis on practical tools, peer-to-peer engagement, and strategic insights, RDDS equips attendees with the knowledge and confidence to shape research agendas and de-risk therapeutic pipelines.

“Rare disease patient advocates are driving drug development efforts like never before, and they need access to tools and training that match their urgency and ambition,” said Charlene Son Rigby, CEO of Global Genes. “RDDS is a transformative experience that equips advocates to translate their lived experience into research readiness and strategic impact for their communities.”

Attendees will explore the drug development journey through a mix of keynote presentations, targeted breakout sessions, expert office hours, and live pitch coaching seminar designed to build actionable research portfolios. Advocates will learn how to:

• Align therapeutic strategies with disease biology to make informed, data-driven research decisions
• Use natural history studies, biomarkers, and patient-reported outcomes to catalyze clinical progress
• De-risk their disease and develop compelling research narratives to attract investment and partnerships
• Engage effectively with clinical research from day one, driving trial readiness and patient-centered design

Confirmed speakers include trailblazing researchers and advocates such as Dr. Wendy Chung, Dr. Tim Yu, and Global Genes CEO Charlene Son Rigby, each of whom has redefined the role of patients in therapeutic development.

The symposium also highlights the growing role of next-generation advocacy and the importance of collaborative, community-driven research efforts like those enabled by the RARE-X program, Global Genes’ patient-owned data collection platform.

The 2024 symposium demonstrated significant impact: 94% of attendees reported increased knowledge of research and drug development; 85% felt more confident addressing their organization’s research goals; and 97% made meaningful connections to accelerate their work.

“RDDS is not just another conference,” added Rigby. “It’s a catalyst for rare disease advocates who are ready to lead, and build vibrant ecosystems to progress work in their disease. Whether you’re initiating your first research project or scaling an existing strategy, this event will give you the clarity, connections, and confidence to take the next step.”

Thank You to Our Sponsors
 This year’s RDDS is made possible with the generous support of our sponsors: Amgen Rare Disease, Alexion Charitable Foundation, Pharming, Travere Therapeutics, UCB, Acadia, Amicus Therapeutics, BioCryst, Bio, BridgeBio, Catalyst Pharmaceutical, Chiesi Global Rare Diseases

To learn more or register, visit: https://globalgenes.org/rare-drug-development-symposium-2025/

Direct Registration Link: https://globalgenes.tfaforms.net/5029232

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 brings together biopharma, researchers, and advocates with data at the core, Global Genes also enables research and data collection through the RARE-X program, building the largest collaborative, patient-driven, open-data access initiative for rare diseases globally.

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SOURCE RARE-X