Rare Disease Day 2024

Orphan Products for Rare Diseases: Availability and Accessibility - They're Not The Same Thing

Thursday, March 7, 2024

Keynote speakers

David Fajgenbaum

David Fajgenbaum, MD, MBA, MSc

Associate Professor of Medicine
Director, Center for Cytokine Storm Treatment & Laboratory
Associate Director, Patient Impact, Orphan Disease Center
University of Pennsylvania
Co-Founder & President, Castleman Disease Collaborative Network
Co-Founder & President, Every Cure


“From Chasing My Cure to Every Cure: Leveraging AI to unlock the full potential of every medicine.”

David McLean

David J. McLean, PhD

Chief Executive Officer of Emerging Therapy Solutions

“The Multiple Challenges in Accessing Orphan Products for Patients, Families and Clinicians”

Congratulations to our Research Award Winners!

  1. Miles Greenberg, St. Olaf College
    Enzyme Replacement is Associated With Better Long-term Cognitive Outcomes in Hurler Syndrome
  2. Katelyn Sawyer, Medical School 
    Assessing Readability of Public Facing Online Materials on Rare Disorders of Fatty Acid Oxidation
  3. Bryan Yan, College of Pharmacy
    What Is Life Like For An Adult Living With Achondroplasia?
  4. Adeboye Bamgboye, College of Pharmacy 
    A Comparison of IV and Oral Topiramate Pharmacokinetics in Healthy Research Participants, Patients with Migraines, and Patients with Epilepsy with or without Concomitant Enzyme-Inducing Drugs


Acadia Pharmaceuticals
Alexion Pharmaceuticals
BioMarin Pharmaceutical Inc.
bluebird bio
Chiesi Global Rare Diseases
Fairview Pharmacy Services
Gillette Children's Specialty Healthcare
Ipsen Biopharmaceuticals
Jazz Pharmaceuticals
Takeda Foundation
Upsher-Smith Laboratories  

If you are interested in learning about Rare Disease Day sponsorship opportunities, please contact Lori Endsley.

Posters & Post-Event Access to Whova

You can still view posters and virtual tables! All event content will remain available to attendees on Whova until 6/7/2024.

Rare Disease Day 2024 share your colors banner. Diverse group of people proudly standing in front of RDD logo.