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Rare disease advocates learn to accelerate therapeutic development at 9th RARE Drug Development Symposium

ALISO VIEJO, Calif., March 14, 2024 /PRNewswire-PRWeb/ — The rare disease patient advocacy organization Global Genes and the Orphan Disease Center at the University of Pennsylvania will host the 9th Annual RARE Drug Development Symposium, an event that provides patient advocates with the knowledge, skills, and connections they need to advance therapeutic development for their communities.

The event brings together patient advocates, biopharmaceutical executives, and researchers who have successfully navigated the therapeutic development process, formed successful research collaborations, and overcome financial and regulatory hurdles for two days of panel discussions, hands-on workshops, and one-on-one meetings with drug development experts.

“Since Global Genes and the Orphan Disease Center launched theRARE Drug Development Symposiumnine years ago, patient advocates have emerged as critical catalysts and drivers of therapeutic development,” said Charlene Son Rigby, CEO of Global Genes. “They are not only funding early-stage research, but increasingly involved in taking the reins of drug development to ensure needed therapies are advanced for their communities.”

This year’s symposium will focus on innovative ideas from next-generation change-makers. Theagenda for this year’s symposiumwill focus on patient-led data initiatives, how to navigate the challenges of conducting clinical trials for small patient populations, and emerging commercial and non-profit financing models. Keynote speakers include Tania Simoncelli, vice president of science in society at the Chan Zuckerburg Initiative, and Dominique Pichard, M.D., Director of the Division of Rare Diseases Research Innovation at NCATS.

Simoncelli will highlight patient advocacy groups that are forging new paths to speed development of therapies for rare diseases. Dr. Pichard will share her vision for creating scalable models that will accelerate translational research in rare disease, and discuss steps that patients, caregivers, researchers, and organizations can take to help move the needle.

The event will also feature a RARE Resource Fair to connect advocates to service providers, and expert office hours that provide advocates an opportunity to schedule one-to-one meetings with experts on topics such as data collection, research strategy, therapy development, and engaging with the U.S. Food and Drug Administration. Appointments will open for registered attendees in March.        

The RARE-X Exchange Forum pre-conference session informs participants interested in learning about current research studies using the RARE-X data platform and how researchers and patient advocates can leverage the platform to advance research in their area of concern.

The RARE Drug Development Symposium will be held Monday, April 29, 2024 through Wednesday, May 1, 2024 at the Sheraton Philadelphia Downtown hotel at 201 N. 17th Street, Philadelphia, Pennsylvania.

Sponsors for this year’s RARE Drug Development Symposium includeAlexionAmgenAmicus TherapeuticsBioCrystBridgeBioOvid Therapeutics, andSanofi Genzyme. For more information,visit our website.

“Rare disease patient communities are active participants in the drug development process,” said Monique Molloy, Executive Director, Orphan Disease Center at the University of Pennsylvania. “They have enormous power and insight to drive the development of new therapies, and this event is designed to bring key stakeholders together so they can share ideas and chart pathways to success.”

About Global Genes
Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of that mission, Global Genes connects, empowers, and inspires the rare disease community to stand up, stand out, and become more effective on their own behalf⁠—helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. Global Genes serves more than 400 million people around the globe, and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE or visit the Resource Hub.

About the Orphan Disease Center
The Orphan Disease Center at the University of Pennsylvania was formed in 2011 to facilitate translational research and therapy development, addressing the prevalence of orphan diseases that affect a marginalized population of individuals internationally. Because each type of orphan disease affects such a small subset of the population, the need for research on and funding for such rare genetic mutations is grossly unmet. The goal of the Orphan Disease Center is to address these growing needs through continued collaboration and partnership, identifying and providing funding sources, and by providing technological and educational resources to academic researchers, biotech, and pharmaceutical companies alike, to foster therapeutic development and innovative rare disease research initiatives.

Media Contact

Lauren Alford, Global Genes, 949-248-7273,

SOURCE Global Genes

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