2025 Agenda
Sunday, October 19, 2025
Monday, October 20, 2025
Tuesday, October 21, 2025
2:00pm - 8:00pm
Registration
Save time Monday morning by picking up your badge early and joining us for a welcome reception.
6:00pm
Welcome Reception
8:00pm
Rare Cancer Coalition Cocktail Reception
7:30am - 8:00am
Conference Registration and Continental Breakfast
8:00am - 8:10am
NORD’s Welcome & Summit Preview
Pamela Gavin, Chief Executive Officer, NORD
8:10am - 8:40am
Young Voices Keynote Panel
Advances in rare disease diagnosis and treatment have particular relevance for young people living with rare diseases. Young advocates will share their stories, challenges and hopes for the future.
Moderator: Mike Porath, Chief Executive Officer and Founder, The Mighty; Executive Director, Dup15q Alliance
- Micah Clayborne, Founder and Chief Executive Officer, Transplant Teenz
- Avery Garrison, Rare Disease Community Member
8:40am - 9:00am
U.S. Food and Drug Administration (FDA) Address (TBD)
9:00am - 9:20am
National Institutes of Health (NIH) Address (TBD)
9:25am - 10:25am
C-Suite Perspectives
Top level executives working in the rare space will share their thoughts on the current landscape for advancing science, addressing cost concerns and ensuring that patients and patient needs are the fundamental driving factor.
Moderator: Kelly Esperias, Chief Strategy & Operations Officer, NORD
- Miguel Fernández Alcalde, President, EMD Serono
- Christophe Arbet-Engels, MD, PhD – Chief Medical Officer, X4 Pharmaceuticals
- Pamela Gavin, Chief Executive Officer, NORD
- Catherine Owen Adams, BSc., Chief Executive Officer, Acadia Pharmaceuticals
10:30am - 11:00am
Networking Break
11:00am - 11:55am
A Fireside Chat with FDA Leadership
FDA senior officials will sit down with NORD’s CEO for a conversation about their hopes and priorities and, in particular, how they view the future of rare disease product development and review.
- Michael Davis, MD, Deputy Director, Center for Drug Evaluation and Reasearch (CDER), FDA
- Michelle Tarver, MD, PhD, Director of Center for Devices and Radiological Health (CDRH), FDA
12:00pm - 12:30pm
Lightning Rounds Poster Presentations
Authors of the top selected poster abstracts will share their key findings.
12:30pm - 1:55pm
Networking Lunch
2:00pm - 3:55pm
Breakout Sessions
ROOM 1
2:00 – 2:10 pm: Community Voices
- Katie Gillick, Rare Community Member
2:10 – 3:05 pm: A New Era for Rare Disease Clinical Trials
This session will explore how technology and innovative trial designs address issues that historically have made clinical trials for rare diseases particularly challenging.
Moderator: John Scott, PhD, Director, Division of Biostatistics, CBER, FDA
- Claire Beggs, Executive Director, Global Regulatory Affairs and Drug Safety, Jazz Pharmaceuticals
- Kaushik Ghosal PhD, Amyloidosis Research Consortium ASPIRE Collaboration
- Nerissa Kreher, MD, MBA, Chief Medical Officer, Alltrna
- Alan M. Miller, MD, PhD, Chief Medical Officer, TD2 Oncology
3:10 – 4:05 pm: A Moral Imperative: Addressing the Challenges of Pediatric R&D
Even though about two-thirds of Americans with rare diseases are children, pediatric R&D lags far behind adult studies. What are the special challenges of pediatric research and how do we address them?
Moderator: Fumihiko Urano, MD, PhD, Samuel E. Schechter Professor in Medicine, Washington University School of Medicine
- Stephanie Fradette, MD, Head of Neuromuscular Development Unit, Biogen
- Dylan Ritter, PhD, Director of Scientific and Clinical Initiatives, Dup15Q Alliance
- Sheila Wilson, Vice President, Clinical Operations and Data Management, Orchard Therapeutics
Room 2
2:00 - 2:10 pm: Community Voices
- Jana Monaco, Rare Caregiver; Virginia Ambassador, NORD Rare Action Network®
2:10 - 3:05 pm: Charting a Path Forward for Newborn Screening
What are the current challenges related to newborn screening and how do we address them? Hear from leading voices in the field as they discuss recent developments in the American newborn screening landscape and actionable strategies for moving forward.
Moderator: Allison Herrity, MPH, Senior Policy Analyst, NORD
- Danaé Bartke, Executive Director, HCU Network America
- Amy Gaviglio, MS, CGC, Founder and Chief Executive Officer, Connetics Consulting LLC
3:10 - 4:05 pm: Rare Disease Communities of Care: Integrating Mental Health Support into Treatment
Rare disease patients and their families face unique mental health challenges that are often overlooked. This session will explore how mental health support can be integrated into the holistic care model for rare disease patients.
Moderator: Jill Pollander, RN, MSN, Vice President of Patient Services, NORD
- Vanessa Acero, LPCC, Licensed Mental Health Therapist
- Ray Merenstein, Parent; Director of NAMI CO; Colorado Rare Disease Advisory Council
- Andres Trevino, Global Head of Patient Advocacy, Chiesi
4:05pm - 4:30pm
Networking Break
4:30pm - 5:40pm
Special Plenary: Curative Therapies — Opportunities and Challenges
This panel of investors and entrepreneurs will share their perspectives on the outlook for rare disease investment in 2026 and beyond.
Moderator: David Scheer, President, Scheer & Company, Inc.
- Martin Mackay, PhD, Co-Founder, RallyBio
- Vinod Mitta, Director, Seamless Care Models Group, CMS Innovation Center, US HHS.
- Sean P. Nolan, Chief Executive Officer, Taysha Gene Therapies
- Maha Radhakrishnan, MD, Executive Partner, Sofinnova Investments
- Philip Ross, Vice Chairman, Jefferies LLC
- Stephen Squinto, PhD, Chief Investment Officer, J.P. Morgan Life Science Private Capital
- David Tischler, Chief Executive Officer, CureTrust
5:30pm
Exhibit Hall and ‘Sip in Science’ Poster Hall Reception
7:30am - 8:15am
Continental Breakfast and Registration
8:15am - 8:45am
Community Voice Keynote: Preparing the Next Generation
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 Grossman, M.Sc., Vice Chair, FOP Germany/IFOPA
- Advait Wattal, Ambassador, NORD Rare Action Network®; Student, University of Pennsylvania
8:50am - 9:45am
Making Your Voice Heard: Rare Disease Advocacy and the 119th Congress
Representatives of the Rare Disease Congressional Caucus will share their thoughts on current and upcoming policy priorities, and how patient advocates can have an impact.
9:50am - 10:45am
Case Studies in Collaboration
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.
- R. Duane Clark, General Manager, U.S. Rare Diseases, Sanofi
- Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D, Agios
- Cynthia Grossman, PhD, Director, Division of Patient-Centered Development, CDRH, FDA
- Terri Klein, CNPM, MPA, President and Chief Executive Officer, National MPS Society
10:45am - 11:15am
Networking Break
11:15am - 12:10pm
How FDA Review Teams Incorporate Patient Data
Panel composed entirely of FDA reviewers sharing their thoughts and firsthand experiences.
- Robyn Bent, MS, RN, Director, Patient-Focused Drug Development, CDER, FDA
12:15pm - 1:25pm
Networking Lunch
1:30pm - 1:40pm
Community Voices: Patient/Caregiver Story
- Lee and Noa Greenwood, Rare Community Members
1:40pm - 2:35pm
What’s Next for Gene and Cell Therapies?
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
- Philip J. Brooks, PhD, Acting Director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), NIH
- Terence Flotte, MD, President, ASGCT
- Mimi Lee, MD, PhD, Program Manager, Health Science Futures, ARPA-H
2:40pm - 3:35pm
The Role of AI in Drug Development
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
- Anindita (Annie) 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 R&DS
3:40pm - 4:35pm
Closing Session: Next Steps for Optimal Impact
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 Greally, MD, Children’s Hospital at Montefiore Einstein
- Amy Rick, JD, Director of Strategic Coalitions, FDA’s Rare Disease Innovation Hub
- 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
4:35pm
Closing Remarks
Pamela Gavin, Chief Executive Officer, NORD
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