Orphan Products for Rare Diseases: Availability and Accessibility - They're Not The Same Thing
Thursday, March 7, 2024
Keynote speakers
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
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 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!
- Miles Greenberg, St. Olaf College
Enzyme Replacement is Associated With Better Long-term Cognitive Outcomes in Hurler Syndrome - Katelyn Sawyer, Medical School
Assessing Readability of Public Facing Online Materials on Rare Disorders of Fatty Acid Oxidation - Bryan Yan, College of Pharmacy
What Is Life Like For An Adult Living With Achondroplasia? - 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
Sponsors
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
Sanofi
Takeda Foundation
Upsher-Smith Laboratories
If you are interested in learning about Rare Disease Day sponsorship opportunities, please contact Lori Endsley.
