World Orphan Drug Congress USA 2019


Wednesday April 10- Friday, April 12
Gaylord National Harbor Hotel, Oxon Hill, Maryland USA

 

THE GLOBAL ORPHAN DRUG CONFERENCE AND EXPO

ONLY 30 PASSES LEFT- BOOK NOW!

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Title Sponsor

 

 
1,200+
Attendees
50+
Countries
350+
Speakers
2100+
Meetings
240+
Sessions

What is the World Orphan Drug Congress USA?

 

World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster.
 

Bringing together a global gathering of 1,200 leaders in orphan drugs from 50 countries, World Orphan Drug Congress USA will feature over 135 presentations covering all aspects of orphan drug development and rare disease research.
 

Together with: 

Rare Disease Advocacy World USA 2019

Pitch & Partner 2019

 

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 350+ Speakers Including

 
John Maraganore at World Orphan Drug Congress 2019
John Maraganore
Chief Executive Officer and Director
Alnylam Pharmaceuticals
Paul Levesque at World Orphan Drug Congress 2019
Paul Levesque
Global President
Pfizer Rare Disease
Emil Kakkis at World Orphan Drug Congress 2019
Emil Kakkis
Chief Executive Officer and President
Ultragenyx
James Greenwood at World Orphan Drug Congress 2019
James Greenwood
Chief Executive Officer
Biotechnology Innovation Organization
Amin Hussain Al Amiri at World Orphan Drug Congress 2019
Amin Hussain Al Amiri
Asst Undersecretary of Public Health Policy & Licensing
United Arab Emirates Ministry of Health and Prevention
Arndt Rolfs at World Orphan Drug Congress 2019
Arndt Rolfs
Chief Executive Officer,
CENTOGENE
Peter Saltonstall at World Orphan Drug Congress 2019
Peter Saltonstall
President and Chief Executive Officer
NORD
Yann Le Cam at World Orphan Drug Congress 2019
Yann Le Cam
Chief Executive Officer
EURORDIS
Michelle Berg at World Orphan Drug Congress 2019
Michelle Berg
Vice President Patient Affairs
Abeona Therapeutics
     Peter Marks at World Orphan Drug Congress 2019
     Peter Marks
Director
Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration
     Linn Parrish at World Orphan Drug Congress 2019
     Linn Parrish
Corporate Responsibility
Shire
     Sandy Macrae at World Orphan Drug Congress 2019
     Sandy Macrae
Chief Executive Officer
Sangamo Therapeutics
     Debra Miller at World Orphan Drug Congress 2019
     Debra Miller
President & CEO
Cureduchenne
     Samit Hirawat at World Orphan Drug Congress 2019
     Samit Hirawat
Executive Vice President, Head of Oncology Global Development
Novartis
     Sarah Pitluck at World Orphan Drug Congress 2019
     Sarah Pitluck
Vice President and Head of Global Pricing and Reimbursement
Spark Therapeutics
     Simon Kos at World Orphan Drug Congress 2019
     Simon Kos
Chief Medical Officer
Microsoft
     Anne Pariser at World Orphan Drug Congress 2019
     Anne Pariser
Director
Office of Rare Disease Research
     Marshall Summar at World Orphan Drug Congress 2019
     Marshall Summar
Director, Rare Disease Institute
Children’s National Health System
     Rick Chapman at World Orphan Drug Congress 2019
     Rick Chapman
Director of Health Economics
ICER
     Mark Rothera at World Orphan Drug Congress 2019
     Mark Rothera
Chief Executive Officer
Orchard Therapeutics
     Jerry Collins at World Orphan Drug Congress 2019
     Jerry Collins
Associate Director, Developmental Therapeutics Program Division of Cancer Treatment and Diagnosis
National Cancer Institute
     André Choulika at World Orphan Drug Congress 2019
     André Choulika
Chief Executive Officer
Cellectis
     Matt Patterson at World Orphan Drug Congress 2019
     Matt Patterson
Chief Executive Officer
Audentes Therapeutics
     Susan McCune at World Orphan Drug Congress 2019
     Susan McCune
Director, Office of Pediatric Therapeutics, Office of the Commissioner
U.S. Food and Drug Administration (FDA)
     Samarth Kulkarni at World Orphan Drug Congress 2019
     Samarth Kulkarni
Chief Executive Officer
CRISPR Therapeutics
     Charlie Albright at World Orphan Drug Congress 2019
     Charlie Albright
Chief Scientific Officer
Editas Medicine
     Julie Tierney at World Orphan Drug Congress 2019
     Julie Tierney
Senior Policy Advisor, Strategic Planning and Legislation
Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration
     Walter Kowtoniuk at World Orphan Drug Congress 2019
     Walter Kowtoniuk
Principal
Third Rock Ventures
     Denise Scots-Knight at World Orphan Drug Congress 2019
     Denise Scots-Knight
CEO and Co-Founder
Mereo Biopharma
     Harsha Rajasimha at World Orphan Drug Congress 2019
     Harsha Rajasimha
Co-Founder
Organization for Rare Diseases, India
     Francis Arickx at World Orphan Drug Congress 2019
     Francis Arickx
Head of the directorate Reimbursement of Medicines and Pharmaceutical Policy
National Institute for Health and Disability Insurance (NIHDI RIZIV/INAMI)<, Belgium
     Ailsa Brown at World Orphan Drug Congress 2019
     Ailsa Brown
Lead Health Economist
Scottish Medicines Consortium
     Max Bronstein at World Orphan Drug Congress 2019
     Max Bronstein
Senior Director, Health Policy & Corporate Affairs
Audentes Therapeutics
     Sheela Upadhyaya at World Orphan Drug Congress 2019
     Sheela Upadhyaya
Associate Director Highly Specialized Technologies Centre for Health Technology Evaluation
NICE
     Amanda Bok at World Orphan Drug Congress 2019
     Amanda Bok
CEO
European Hemophilia Consortium
     Philip J Brooks at World Orphan Drug Congress 2019
     Philip J Brooks
Program Director, Office of Rare Diseases Research and Division of Clinical Innovation
NCATS/NIH
     Detlev Parow at World Orphan Drug Congress 2019
     Detlev Parow
Head of Patient Care Management
DAK-Gesundheit
     Janet Rae at World Orphan Drug Congress 2019
     Janet Rae
Vice President, Global Head, Gene Therapy Regulatory Affairs
Ultragenyx Pharmaceutical
     Derek Adams at World Orphan Drug Congress 2019
     Derek Adams
Chief Technology and Manufacturing Officer
Bluebird Bio
     Dan Tierno at World Orphan Drug Congress 2019
     Dan Tierno
Global Data Sciences and Analytics
Bayer US LLC
     James Wilson at World Orphan Drug Congress 2019
     James Wilson
Director, Gene Therapy Program
Orphan Disease Center, University of Pennsylvania School of Medicine
     Roser Bordas Francisco at World Orphan Drug Congress 2019
     Roser Bordas Francisco
Head Technician, Rare Diseases Program of Catalonia
CatSalut
     John Evans at World Orphan Drug Congress 2019
     John Evans
Chief Executive Officer
Beam Therapeutics
     Kevin Huang at World Orphan Drug Congress 2019
     Kevin Huang
President
Chinese Organization for Rare Disorders
     Paul Melmeyer at World Orphan Drug Congress 2019
     Paul Melmeyer
Director of Federal Policy
National Organization for Rare Diseases
     Melissa Hogan at World Orphan Drug Congress 2019
     Melissa Hogan
Founder, President
Project Alive
     Christopher Austin at World Orphan Drug Congress 2019
     Christopher Austin
Director
National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
     Clark Paramore at World Orphan Drug Congress 2019
     Clark Paramore
Head of Value Demonstration
bluebird bio
     Alexander Natz at World Orphan Drug Congress 2019
     Alexander Natz
Secretary General
European Confederation of Pharmaceutical Entrepreneurs (Belgium)
     Ilan Irony at World Orphan Drug Congress 2019
     Ilan Irony
Deputy Director Division of Clinical Evaluation and Pharmacology
Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration
     Vikram Karnani at World Orphan Drug Congress 2019
     Vikram Karnani
Chief Commercial Officer
Horizon Pharma
     Michael Cox at World Orphan Drug Congress 2019
     Michael Cox
Senior Director of Clinical Development and Medical Affairs
Loxo Oncology
     Jeff Allen at World Orphan Drug Congress 2019
     Jeff Allen
President and Chief Executive Officer
Friends of Cancer Research
     Alexander Cole at World Orphan Drug Congress 2019
     Alexander Cole
Global Head, Epidemiology
Alexion Pharmaceuticals
     Sophie Schmitz at World Orphan Drug Congress 2019
     Sophie Schmitz
Managing Partner
Partners4Access
     Daniel Anderson at World Orphan Drug Congress 2019
     Daniel Anderson
Head of Commercial Partnerships
Invitae
     Stephen Hyde at World Orphan Drug Congress 2019
     Stephen Hyde
Principle Investigator
Oxford University, Cystic Fibrosis Gene Therapy Consortium (GTC)
     Margaret Vernon at World Orphan Drug Congress 2019
     Margaret Vernon
Vice President, General Manager Patient-Centered Research
Evidera - Evidence, Value & Access by PPD
     Stuart Bell at World Orphan Drug Congress 2019
     Stuart Bell
Vice President Consulting
Inceptua Medicines Access
     Jim Lang at World Orphan Drug Congress 2019
     Jim Lang
Chief Executive Officer
EVERSANA

 

VIEW ALL 200+ SPEAKERS

2019 Sponsors & Exhibitors

 

  • Title Sponsor

    CENTOGENE AG at World Orphan Drug Congress USA 2019

    Platinum Sponsor

    Eversana at World Orphan Drug Congress USA 2019

    Platinum Sponsor

    Inceptua at World Orphan Drug Congress USA 2019

  • Gold Sponsor

    Clinigen Group at World Orphan Drug Congress USA 2019

    Gold Sponsor

    McKesson Specialty Health at World Orphan Drug Congress USA 2019

    Gold Sponsor

    HVH Precision Analytics at World Orphan Drug Congress USA 2019

  • Gold Sponsor

    Invitae at World Orphan Drug Congress USA 2019

    Gold Sponsor

    Pfizer at World Orphan Drug Congress USA 2019

    Gold Sponsor

    Orchard Therapeutics at World Orphan Drug Congress USA 2019

  • Gold Sponsor

    Partners4Access at World Orphan Drug Congress USA 2019

    Gold Sponsor

    PPD at World Orphan Drug Congress USA 2019

    Gold Sponsor

    Premier Research at World Orphan Drug Congress USA 2019

  • Gold Sponsor

    Syneos Health at World Orphan Drug Congress USA 2019

    Patient Advocacy Sponsor

    National Organization for Rare Disorders (NORD) at World Orphan Drug Congress USA 2019

    Patient Advocacy Sponsor

    EURORDIS at World Orphan Drug Congress USA 2019

  • Patient Advocacy Sponsor

    Rare Disease International at World Orphan Drug Congress USA 2019

    Silver Sponsor

    Clincierge at World Orphan Drug Congress USA 2019

    Silver Sponsor

    Engage Health at World Orphan Drug Congress USA 2019

  • Silver Sponsor

    Huron Consulting Group at World Orphan Drug Congress USA 2019

    Silver Sponsor

    Medvance at World Orphan Drug Congress USA 2019

    Silver Sponsor

    MNG Laboratories at World Orphan Drug Congress USA 2019

  • Silver Sponsor

    myTomorrows at World Orphan Drug Congress USA 2019

    Silver Sponsor

    Orphan Pacific at World Orphan Drug Congress USA 2019

    Silver Sponsor

    PAREXEL International at World Orphan Drug Congress USA 2019

  • Silver Sponsor

    PRA Health Sciences at World Orphan Drug Congress USA 2019

    Silver Sponsor

    rareLife solutions at World Orphan Drug Congress USA 2019

    Silver Sponsor

    TrialCard at World Orphan Drug Congress USA 2019

  • Silver Sponsor

    Worldwide Clinical Trials at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Ambry Genetics at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    AnovoRx Group Llc at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    BluePrint Orphan at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Catalent Pharma Solutions at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Aeglea Biotherapeutics at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    Covance at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Cydan II, Inc. at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Dolon at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    Durbin at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Early Access Care at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Germfree Laboratories Inc at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    IPM.ai at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Lapidus Data at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Medical Marketing Economics at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    Rare Disease Research Center at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Rho, Inc at World Orphan Drug Congress USA 2019

    Bronze Sponsor

    Synteract at World Orphan Drug Congress USA 2019

  • Bronze Sponsor

    VOZ Advisors at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    Baebies at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    BioSensics LLC at World Orphan Drug Congress USA 2019

  • RARE Technology Sponsor

    Droice Labs at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    Deep 6 AI at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    OpenApp at World Orphan Drug Congress USA 2019

  • RARE Technology Sponsor

    RDMD Inc. at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    TREND Community at World Orphan Drug Congress USA 2019

    RARE Technology Sponsor

    Backpack Health at World Orphan Drug Congress USA 2019

 

 

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