Keynote Sessions

 

Keynote, Wednesday 24 April 2024

Panel discussion
08:55

Expediting drug development for rare disease

Moderator: Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Panel discussion
09:45

The innovator dilemma, a call to action

Alaa Hamed, Global Head, Medical Affairs Rare Disease and Rare Blood Disorders, Sanofi
Panel discussion
10:05

Challenge and opportunity with the current regulatory and legislative landscape in the US

Kinnari Patel, President & COO, Rocket Pharma
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center
Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases
Panel discussion
Emil Kakkis
16:45

Using biomarkers to enable successful development of disease-modifying therapies for rare disease

Patricia Weltin
17:40

Portrait unveiling by Beyond the Diagnosis

17:45

COCKTAIL RECEPTION

Join us for cocktail hour

last published: 20/Apr/24 16:35

Keynote, Thursday 25 April 2024

Panel discussion
Panel discussion
10:00

Unlocking access: Transformative policies and strategies for orphan drug accessibility

Moderator: Jayson Slotnik, Partner, Health Policy Strategies, LLC
Christoph Glaetzer, Chief Global Value and Access Officer, Johnson & Johnson
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
Panel discussion
Panel discussion
16:35

WORLD ORPHAN DRUG CONGRESS CONCLUDES

Mark your calendars for World Orphan Drug Congress 2025!

  • Date: April 23-25, 2025
  • Location: Boston Convention and Exhibtion Center (BCEC)
  • If interested in speaking reach out to gina.mchugh@terrapinn.com
  • If interested in sponsoring reach out to justin.franks@terrapinn.com
last published: 20/Apr/24 16:35

 

Get Involved At World Orphan Drug Congress USA

 

To Sponsor Or Exhibit

 
 

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

 

 

 

To Speak

 
 

Gina McHugh
gina.mchugh@terrapinn.com
t/ +1 646 619 1775

 

 

 

Marketing & Press Opportunities

 
 

Kaylie Brogan
kaylie.brogan@terrapinn.com