Global Genes and Boston Children’s Hospital Partner for 2025 RARE Drug Development Symposium
June 24, 2025
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, D.C.—June 24, 2025
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.”
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 a 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. TimYu, and Global Genes CEO Charlene SonRigby, 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.”
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 this page.
Direct Registration Link: https://globalgenes.tfaforms.net/5029232
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 this page.
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.
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.