The International Rare Diseases Research Consortium will be at the forefront of discussions at World Orphan Drug Congress USA 2026, highlighting innovations in rare disease research, personalized therapies, and sustainable approaches to genetic medicine. Attendees should not miss these pivotal sessions:
10 June, 11:30 – Where is N-of-1 Going? | Advanced Therapies – Fireside Chat
Moderated by Dr. David Pearce, Chair of IRDiRC, this session explores the evolving field of N-of-1 therapies for ultra-rare diseases. Speakers:
- Sarah Glass, Chief Operating Officer, n-Lorem Foundation
- Timothy Yu, Attending Physician and Investigator, Boston Children’s Hospital
10 June, 14:30 – IRDiRC Task Force: Facilitating the Development of Preventative Medicines for Rare Diseases
Led by Jon Brudvig, Senior Director of Scientific Evidence at Amicus Therapeutics, this session focuses on strategies to advance preventative therapies for rare diseases through robust clinical development and regulatory pathways.
10 June, 16:40 – Beyond N-of-1: Sustainability of Genetic Therapies for Rare Diseases | Keynote Session
Moderated by Dr. David Pearce, this session examines the long-term scientific, economic, and ethical sustainability of genetic therapies, moving from individualized N-of-1 treatments to scalable and equitable models of care. Speakers include:
- P.J. Brooks, Deputy Director, Division of Rare Diseases Research Innovation, NIH/NCATS
- Alexander Natz, Secretary General, EUCOPE
- Karen Kahn, Co-founder, ForeBatten Foundation
11 June, 14:00 – IRDiRC Task Force: Bridging Diagnostics to Care for Rare Diseases
Lisa Emrick, Medical Director of Neurogenetics at Baylor College of Medicine, will discuss strategies for linking diagnosis to therapy and comprehensive care for patients with rare diseases.
Congress Details:
- Full Agenda
- Registration: https://secure.terrapinn.com/V5/step1.aspx?E=11031&p=1
- Dates: 9–11 June 2026
- Location: Thomas M. Menino Convention & Exhibition Center, Boston, MA, USA