April 14 – April 15, 2026
Arlington, VA

NORD® Rare Disease Scientific Symposium
Advancing Innovation in Rare Disease Research

April 14 – 15, 2026 | Arlington, VA

Register Now

NORD® Rare Disease Scientific Symposium

Rare disease breakthroughs don’t happen in isolation—they require collaboration, innovation, and a united commitment to progress. That’s why the NORD® Rare Disease Scientific Symposium is bringing together a dynamic community of researchers, clinicians, industry leaders, and advocates to spark the conversations and connections that drive science forward.

This immersive, 1.5 day event is designed to push boundaries and bridge gaps across the rare disease landscape. Through engaging case studies, interactive panel discussions, and extended audience Q&A, participants will explore critical topics such as:

  • Innovative Clinical Trial Designs
  • FDA Regulatory Innovation: Plausible Mechanism and Platform Pathways
  • Abandoned, Shelved, and Rescued Therapies for Ultra-Rare Diseases
  • Using Alternative and Real-World Data for Rare Disease Research
  • Access and Coverage: Data and Clinical Development
  • Plus, a Keynote Address from NIH Director Dr. Jay Bhattacharya

Let’s break barriers and build progress—together.

Meet Our Medical & Scientific Trailblazer Honorees

NORD is proud to honor Dr. Susan Berry and Dr. Stephen Kingsmore as Rare Impact Award® recipients for their transformative impact on rare disease care and innovation.

Medical & Scientific Trailblazer

Susan A. Berry, MD

University of Minnesota, a NORD Rare Disease Center of Excellence

Dr. Susan Berry is a nationally recognized leader in medical genetics whose work has transformed care for people with rare diseases across clinical practice, research, and public health. At the University of Minnesota, she helped pioneer coordinated, lifelong care models and led major initiatives like the IBEM-IS registry, advancing understanding and outcomes for inherited metabolic disorders. A dedicated advocate and mentor, her influence has shaped newborn screening, patient-centered care, and rare disease policy nationwide.

Medical & Scientific Trailblazer

Stephen Kingsmore, MD, DSc

Rady Children’s Institute for Genomic Medicine, a NORD Rare Disease Center of Excellence

Dr. Stephen Kingsmore is a pioneering physician-scientist whose work has transformed the diagnosis and treatment of rare genetic diseases, helping bring precision medicine into clinical care. A leader in rapid whole genome sequencing, he advanced life-saving genetic testing for critically ill infants and developed platforms like BeginNGS to expand newborn screening and treatment at scale. His innovations, mentorship, and advocacy have significantly broadened access to genomic medicine for children and families worldwide.

Why Attend

Stay Ahead

Learn about emerging research methodologies, data sources, and case studies that can benefit all rare disease research.

Engage

Join thought-provoking discussions with regulators and leaders in rare disease research and clinical practice.

Adapt & Innovate

See how research strategies and collaborations can fortify and sustain your work in a rapidly shifting landscape.

Conference Venue

The 2026 NORD Rare Disease Scientific Symposium will take place at the Renaissance Arlington Capital View Hotel.

Renaissance Arlington Capital View Hotel
2800 South Potomac Avenue Arlington, VA 22202

NORD has arranged a block of rooms at the Renaissance Arlington Capital View Hotel with the reduced rate of $299 per night on the nights of April 13 and April 14.

Deadline to book is Monday, March 30, 2026.

Book Your Stay

Registration Information

Your registration for this in-person event includes the following:

  • Event Sessions: Access to all general sessions during the event
  • Meals: Enjoy breakfast, snacks, and lunch during the Symposium
  • Receptions: Attend the conference Welcome Reception
  • Networking Opportunities: Ample opportunities to connect with thought leaders, sponsors, exhibitors, and other stakeholders throughout the event

If you are a member of the media, please contact [email protected] to register.

Meet Our Advisory Committee

Annette Bakker, PhD

Chief Executive Officer

Children's Tumor Foundation

Jennifer Cohen, MD

Assistant Professor of Pediatrics, Division of Medical Genetics

Duke University School of Medicine

Christal Delagrammatikas, PhD

Director of Research

Malan Syndrome Foundation

Ravi R. Pathak, PhD, MBA

Medical Director, Lysosomal Storage Disorders, US Medical

Takeda Pharmaceutical Company

Angela Waanders, MD, MPH, MS

Section Head, Neuro-Oncology

Ann and Robert H. Lurie Children’s Hospital of Chicago

Susan Rhee, MD

Executive Medical Director, Clinical Sciences

Regeneron

Giovanni Passiatore, Ph.D.

Head of North America Medical Affairs

Chiesi Global Rare Disease