2025 Agenda

Four students will share how their personal connection to rare has shaped their career plans.

Moderator: Sheldon Schuster, PhD, Professor and President Emeritus, Keck Graduate Institute; Vice Chair, NORD Board of Directors

• Brooke Foster, Student, Rider University 
• Brooke Friedman, BA, Neuroscience PhD Student, Icahn School of Medicine at Mt. Sinai; New York Ambassador, NORD Rare Action Network®
• Nadine Groβmann, M.Sc., Vice Chair, FOP Germany/IFOPA
• Advait Wattal, Ambassador, NORD Rare Action Network®; Student, University of Pennsylvania 

This session will explore current federal and state policy issues impacting the rare disease community, recent legislative developments and strategies for effective engagement with policymakers.

Moderator: Jay Keese, Principal, CEO, Capitol Advocates, LLC

• Leigh Ann Fairley, Health Legislative Assistant, U.S. Representative Gus Bilirakis (FL-12)
• Carolyn Sheridan, MPH, Associate Director of State Policy, NORD
• Jackie Weinrich, Senior Health Policy Advisor, U.S. Representative Doris Matsui (CA-07)

Shelley Bowen, Director, Family Services and Advocacy, Barth Syndrome Foundation

When patient organizations and companies work together in ways that are ethical, practical and creative, patients and families benefit. This session will look at case studies of collaboration and the best practices they illustrate.

Moderator: Cynthia Grossman, PhD, Director, Division of Patient-Centered Development, CDRH, FDA

• Kevin Cancelliere, Senior Associate Director, Patient Marketing, LEO Pharma
• R. Duane Clark, General Manager, U.S. Rare Diseases, Sanofi
• Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D, Agios Pharmaceuticals
• Leah M. Howard, JD, President and CEO, National Psoriasis Foundation
• Golie-Lorenzo Green, Rare Community Member
• Terri Klein, CNPM, MPA, President and CEO, National MPS Society

Panel composed entirely of FDA reviewers sharing their thoughts and firsthand experiences.

Moderator: Robyn Bent, MS, RN, Director, Patient-Focused Drug Development, CDER, FDA

• Theresa Bourne, MD, Medical Officer, Division of Rare Diseases and Medical Genetics, CDER, FDA
• Naomi Knoble, PhD, Associate Director, Division of Clinical Outcome Assessment, CDER, FDA

• Lee and Noa Greenwood, Rare Community Members

Co-hosted by NORD and the American Society of Gene and Cell Therapies (ASGCT), this panel will discuss the current state-of-the-art, advances on the horizon and policy and economic challenges.

Moderator: Edward Neilan, MD, PhD, Chief Medical and Scientific Officer, NORD

• Jennifer Adair, PhD, FUNAS, Professor and Vice Chair, Department of Genetic & Cellular Medicine, UMASS Chan Medical School
• Terence Flotte, MD, Provost, Executive Deputy Chancellor and Dean, UMass Chan Medical School; President, ASGCT
• Mimi Lee, MD, PhD, Program Manager, Health Science Futures, ARPA-H

How can we harness the power of AI to expedite rare disease drug development, while still ensuring safety and efficacy? This panel of experts will explore the many ways AI is being utilized to accelerate and transform drug discovery.

Moderator: Tracey Sikora, Vice President of Research and Clinical Programs, NORD

• Bruce Bloom, JD, DDS, Rare Disease Community Liaison, Healx
• Tina Hu-Rodgers, Co-Leader, Life Sciences Industry Group, Buchanan Ingersoll & Rooney
• Anindita Saha, Associate Director for Data Science and Artificial Intelligence Policy (acting), Office of Medical Policy, CDER, FDA
• Jian Wang, PhD, Vice President, Digital Innovation, IQVIA Research and Development Solutions

NORD’s senior leadership and community thought leaders will share perspectives on current priorities, next steps, and how to have optimal impact together in science, public policy, and patient care.

Moderator: Pamela Gavin, Chief Executive Officer, NORD

• John M. Greally, DMed, PhD, FACMG, Director, New York Center for Rare Diseases, a NORD® Rare Disease Center of Excellence
• Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations, Takeda
• Theresa Strong, PhD, Director of Research Programs, Foundation for Prader-Willi Research

Pamela Gavin, Chief Executive Officer, NORD