2019 speakers

 

Derek Adams, Chief Technology and Manufacturing Officer, bluebird bio

Derek Adams at World Orphan Drug Congress USA 2019

Amin Alamiri, Asst. Undersecretary of Public Health Policy & Licensing, United Arab Emirates Ministry of Health and Prevention

Amin Alamiri at World Orphan Drug Congress USA 2019

Charlie Albright, Chief Commercial Officer, Editas Medicine

Charlie Albright at World Orphan Drug Congress USA 2019

Jeff Allen, President & CEO, Friends of Cancer Research

Jeff Allen at World Orphan Drug Congress USA 2019

Daniel Anderson, Head of Commercial Partnerships, Invitae

Daniel Anderson at World Orphan Drug Congress USA 2019

Karen Anderson, Senior Director - Medical Affairs & Rare Genetic Diseases, Agios Pharmaceuticals

Karen Anderson at World Orphan Drug Congress USA 2019

Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy,, National Institute for Health and Disability Insurance RIZIV-INAMI

Francis Arickx at World Orphan Drug Congress USA 2019

Jamie Arnott, Senior Project Director, Rho, Inc

Jamie Arnott at World Orphan Drug Congress USA 2019

Christopher Austin, Director, National Center for Advancing Translational Sciences (NCATS), National Center for Advancing Translational Sciences - NCATS

Christopher Austin at World Orphan Drug Congress USA 2019

Imran Babar, Chief Business Officer, Cydan II, Inc.

Imran Babar at World Orphan Drug Congress USA 2019

Melinda Bachini, Advocacy Coordinator, The Cholangiocarcinoma Foundation

Melinda Bachini at World Orphan Drug Congress USA 2019

Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals

Mark Baglin at World Orphan Drug Congress USA 2019

Rahul Ballal, Cheif Executive Officer, Imara

Rahul Ballal at World Orphan Drug Congress USA 2019

Mariah Baltezegar, Executive Director Clinical Development, Syneos Health

Mariah Baltezegar at World Orphan Drug Congress USA 2019

Michael Barry, Clinical Director National Centre for Pharmacoeconomics, St James's Hospital

Michael Barry at World Orphan Drug Congress USA 2019

Ron Bartek, President, Director, and Founder, Friedreich's Ataxia Research Alliance

Ron Bartek at World Orphan Drug Congress USA 2019

Heather Behanna, Principal, Sofinnova Ventures

Heather Behanna at World Orphan Drug Congress USA 2019

Stuart Bell, Vice President, Consulting, Inceptua

Stuart Bell at World Orphan Drug Congress USA 2019

Michelle Berg, VP Patient Affairs and Community Engagement, Abeona Therapeutics Inc.

Michelle Berg at World Orphan Drug Congress USA 2019

Nirjal Bhattarai, Principal Investigator, F.D.A. U.S. Food and Drug Administration

Nirjal Bhattarai at World Orphan Drug Congress USA 2019

Amanda Bok, Chief Executive Officer, European Haemophillia Cosortium

Amanda Bok at World Orphan Drug Congress USA 2019

Tim Boyd, Director of State Policy, National Organization for Rare Disorders

Tim Boyd at World Orphan Drug Congress USA 2019

Alfred Boyle, Senior Vice President Technical Operations, Alnylam Pharmaceuticals

Alfred Boyle at World Orphan Drug Congress USA 2019

Aoife Brennan, Chief Executive Officer, Synlogic Inc

Aoife Brennan at World Orphan Drug Congress USA 2019

Brian Bronk, Head of External Innovation, Rare Diseases, Sanofi

Brian Bronk at World Orphan Drug Congress USA 2019

Max Bronstein, Senior Director, Health Policy And Corporate Affairs, Audentes Therapeutics

Max Bronstein at World Orphan Drug Congress USA 2019

Philip Brooks, Program Director, National Center for Advancing Translational Sciences - NCATS

Philip Brooks at World Orphan Drug Congress USA 2019

Philip Brooks, Program Director, National Center for Advancing Translational Sciences - NCATS

Philip Brooks at World Orphan Drug Congress USA 2019

Ailsa Brown, Lead Health Economist, Scottish Medicines Consortium

Ailsa Brown at World Orphan Drug Congress USA 2019

Anne Bruns, Executive Director, The Atypical HUS Foundation

Anne Bruns at World Orphan Drug Congress USA 2019

Alan Cash, Chief Executive Officer and Co-Founder, MetVital

Alan Cash at World Orphan Drug Congress USA 2019

Rick Chapman, Director of Health Economics, Institute for Clinical and Economic Review

Rick Chapman at World Orphan Drug Congress USA 2019

Maurizio Chiriva, Cheif Executive Officer, Kiromic Llc

Maurizio Chiriva at World Orphan Drug Congress USA 2019

Andre Choulika, Chairman & Chief Executive Officer, Cellectis

Andre Choulika at World Orphan Drug Congress USA 2019

Christopher Chute, Bloomberg Distinguished Professor of Health Informatics, Johns Hopkins University

Christopher Chute at World Orphan Drug Congress USA 2019

Jerry Collins, Associate Director, Developmental Therapeutics Program Division of Cancer Treatment and Diagnosis,, National Cancer Institute

Jerry Collins at World Orphan Drug Congress USA 2019

Linda Cooper, Director of Special Initiatives, Association For Creatine Deficiencies

Linda Cooper at World Orphan Drug Congress USA 2019

Michael Cox, Senior Director of Clinical Development and Medical Affairs, Loxo Oncology Inc

Michael Cox at World Orphan Drug Congress USA 2019

Stewart Craig, Chief Manufacturing Officer, Orchard Therapeutics

Stewart Craig at World Orphan Drug Congress USA 2019

Jeremy Cress, Director of Operations, Fibrocell Therapeutics

Brad Crittenden, Executive Director, Canadian Association of Pompe

Brad Crittenden at World Orphan Drug Congress USA 2019

Anne Cropp, Chief Scientific Officer, Early Access Care

Anne Cropp at World Orphan Drug Congress USA 2019

Martin Devenport, Chief Operating Officer, OncoImmune Inc

Martin Devenport at World Orphan Drug Congress USA 2019

Lisa Dilworth, Vice President Rare and Orphan Diseases, Synteract

Lisa Dilworth at World Orphan Drug Congress USA 2019

Johan Drott, Chief Executive Officer, Respiratorius

Johan Drott at World Orphan Drug Congress USA 2019

Kelly Du Plessis, Chief Executive Officer, Rare Diseases South Africa

Kelly Du Plessis at World Orphan Drug Congress USA 2019

Ashish Dugar, Vice President, Global Medical Affairs, Sarepta Therapeutics

Ashish Dugar at World Orphan Drug Congress USA 2019

Tim Dyer, Chief Executive Officer, Addex Therapeutics

Tim Dyer at World Orphan Drug Congress USA 2019

Kara Eichelkraut, Senior Manager, Patient Advocacy, Reata Pharmaceuticals

Kara Eichelkraut at World Orphan Drug Congress USA 2019

Jaysson Eicholtz, Director of GMP Operations, Nationwide Children's Hospital

Jaysson Eicholtz at World Orphan Drug Congress USA 2019

Wendy Erler, VP of Patient Advocacy and Market Insights, Wave Life Sciences

Wendy Erler at World Orphan Drug Congress USA 2019

John Evans, Chief Executive Officer, Beam Therapeutics

John Evans at World Orphan Drug Congress USA 2019

Beata Ferencz, Project Manager, Rare Diseases Sweden

Beata Ferencz at World Orphan Drug Congress USA 2019

Vincent Forster, Chief Executive Officer, Versantis

Vincent Forster at World Orphan Drug Congress USA 2019

Roser Francisco Bordas, Head Technician, Rare Disease Program, Catalan Health Service. Spain, CatSalut

Roser Francisco Bordas at World Orphan Drug Congress USA 2019

John Freshley, President and Chief Executive Officer, ONL Therapeutics

John Freshley at World Orphan Drug Congress USA 2019

Sarah Fry, Global Business Development Manager, Inceptua Medicines Access

Sarah Fry at World Orphan Drug Congress USA 2019

Ilan Ganot, Founder and Chief Executive Officer, Solid GT, LLC

Ilan Ganot at World Orphan Drug Congress USA 2019

Deborah Gill, Professor, Oxford University Press

Deborah Gill at World Orphan Drug Congress USA 2019

Manijeh Goldberg, Chief Executive Officer, Privo Technologies

Manijeh Goldberg at World Orphan Drug Congress USA 2019

Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy

Marianne Hamilton Lopez at World Orphan Drug Congress USA 2019

Dorit Harati, Vice President Of Quality Assurance, Gamida Cell Ltd

Dorit Harati at World Orphan Drug Congress USA 2019

Eric Hartman, Executive Director, Choroideremia Research Foundation Inc

Eric Hartman at World Orphan Drug Congress USA 2019

Melissa Hogan, President, Project Alive

Melissa Hogan at World Orphan Drug Congress USA 2019

John Hopper, President and Executive Director, Fibrolamellar Cancer Foundation

John Hopper at World Orphan Drug Congress USA 2019

Kevin Huang, President, Chinese Organization for Rare Disorders

Kevin Huang at World Orphan Drug Congress USA 2019

Wills Hughes-Wilson, Head of Patient Access & Commercial Planning, Mereo BioPharma

Wills Hughes-Wilson at World Orphan Drug Congress USA 2019

Usman Iqbal, Vice President, Medical Affairs, Acer Therapeutics

Usman Iqbal at World Orphan Drug Congress USA 2019

Ilan Irony, Deputy Director, Food and Drug Administration

Ilan Irony at World Orphan Drug Congress USA 2019

Eugean Jiwanmall, Senior Research Analyst, Technology Evaluation & Medical Policy, Independence Blue Cross

Eugean Jiwanmall at World Orphan Drug Congress USA 2019

Vikram Karnani, Executive Vice President, Chief Commercial Ofiicer, Horizon Pharma

Vikram Karnani at World Orphan Drug Congress USA 2019

Vipul Kashyap, Chief Information Architect, Northwell Health

Vipul Kashyap at World Orphan Drug Congress USA 2019

Manohar Katakam, Founder And Chief Executive Officer, Sterotherapeutics

Manohar Katakam at World Orphan Drug Congress USA 2019

Annie Kennedy, Senior Vice President of Legislation and Public Policy, Parent Project Muscular Dystrophy

Annie Kennedy at World Orphan Drug Congress USA 2019

Alastair Kent, Ambassador, Genetic Alliance U.K.

Alastair Kent at World Orphan Drug Congress USA 2019

Behrang Kianzad, Faculty, University of Copenhagen

Behrang Kianzad at World Orphan Drug Congress USA 2019

Terri Klein, President and CEO, National M.P.S. Society

Terri Klein at World Orphan Drug Congress USA 2019

Simon Kos, Chief Medical Officer, Microsoft

Simon Kos at World Orphan Drug Congress USA 2019

Walter Kowtoniuk, Principle, Third Rock Ventures, LLC

Walter Kowtoniuk at World Orphan Drug Congress USA 2019

Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

Samarth Kulkarni at World Orphan Drug Congress USA 2019

Steven Laffoon, Founder and President, Wylder Nation

Steven Laffoon at World Orphan Drug Congress USA 2019

David Lapidus, President, Lapidus Data

David Lapidus at World Orphan Drug Congress USA 2019

Yann Le Cam, Chief Executive Officer, EURORDIS

Yann Le Cam at World Orphan Drug Congress USA 2019

Kimberly LeBlanc, Associate Director of Research Operations Undiagnosed Diseases Network Coordinating Center, Harvard Medical School

Kimberly LeBlanc at World Orphan Drug Congress USA 2019

Ray Lee, Founder and Chief Medical Officer, Teclison Limited

Ray Lee at World Orphan Drug Congress USA 2019

Thomas Lester, Senior Director of Product Development, BioMarin Pharmaceutical Inc.

Thomas Lester at World Orphan Drug Congress USA 2019

Sook Yee Lim, Co-founder and President, We CARE Journey

Sook Yee Lim at World Orphan Drug Congress USA 2019

Julie Lin, Global Project Head, Early Clinical and Business Development, Sanofi Genzyme Corp

Julie Lin at World Orphan Drug Congress USA 2019

Allyson Lock, NEW ZEALAND POMPE NETWORK

Allyson Lock at World Orphan Drug Congress USA 2019

Robert Long, Executive Director of Strategic Development, Uplifting Athletes

Robert Long at World Orphan Drug Congress USA 2019

Isabelle Lousada, President and CEO, Amyloidosis Research Consortium

Isabelle Lousada at World Orphan Drug Congress USA 2019

Patrick Lu, President and Chief Executive Officer, Sirnaomics, Inc.

Patrick Lu at World Orphan Drug Congress USA 2019

Sanjeev Luther, Chief Executive Officer, Rafael Pharmaceuticals

Sanjeev Luther at World Orphan Drug Congress USA 2019

Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics

Sandy Macrae at World Orphan Drug Congress USA 2019

John Maraganore, Chief Executive Officer, Alnylam Pharmaceuticals

John Maraganore at World Orphan Drug Congress USA 2019

Peter Marks, Director, Biologics Evaluation And Research, Food And Drug Administration UK

Peter Marks at World Orphan Drug Congress USA 2019

Kevin Marsh, Senior Research Scientist and European Director of Modelling, Evidera

Kevin Marsh at World Orphan Drug Congress USA 2019

Tara Mathiesen, Director, US Diagnostics, Orchard Therapeutics

Tara Mathiesen at World Orphan Drug Congress USA 2019

Susan Mccune, Director, Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration/HSS

Susan Mccune at World Orphan Drug Congress USA 2019

Jenn Mcnary, Patient advisory lead, Early Access Care

Jenn Mcnary at World Orphan Drug Congress USA 2019

Paul Melmeyer, Associate Director of Public Policy, National Organization for Rare Disorders

Paul Melmeyer at World Orphan Drug Congress USA 2019

Debra Miller, Founder and President, CureDuchenne

Debra Miller at World Orphan Drug Congress USA 2019

Chris Moen, President, Choroideremia Research Foundation Inc

Chris Moen at World Orphan Drug Congress USA 2019

Juliet Moritz, Executive Director, Program Strategy, Rare Disease & Pediatrics/Patient Engagement, Premier Research

Juliet Moritz at World Orphan Drug Congress USA 2019

Trista Morrison, Vice President of Communications and Patient Advocacy, North America, Sobi

Trista Morrison at World Orphan Drug Congress USA 2019

David Mott, General Partner, N.E.A.

David Mott at World Orphan Drug Congress USA 2019

Michael Murphy, Chief Medical & Scientific Officer, Worldwide Clinical Trials

Michael Murphy at World Orphan Drug Congress USA 2019

Anant Murthy, Vice President of Market Access & Pricing, Alnylam

Anant Murthy at World Orphan Drug Congress USA 2019

Joseph Musumeci, President, BluePrint Orphan

Joseph Musumeci at World Orphan Drug Congress USA 2019

Francois Nader, Board Chairman, Acceleron Pharma (XLRN), Former President & Chief Executive Officer,, NPS Pharmaceuticals Inc

Francois Nader at World Orphan Drug Congress USA 2019

Alexander Natz, Secretary General, EUCOPE

Alexander Natz at World Orphan Drug Congress USA 2019

Knut Niss, Chief Technology Officer, MustangBio

Knut Niss at World Orphan Drug Congress USA 2019

Megan O'Boyle, Principal Investigator, Phelan-McDermid Syndrome Foundation

Megan O'Boyle at World Orphan Drug Congress USA 2019

Jim Palma, Executive Director, TargetCancer Foundation

Jim Palma at World Orphan Drug Congress USA 2019

Clark Paramore, Head Of Value Demonstration, bluebird bio

Clark Paramore at World Orphan Drug Congress USA 2019

Anne Pariser, Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences - NCATS

Anne Pariser at World Orphan Drug Congress USA 2019

Detlev Parow, Head of Department of Medicines, Therapeutic Appliances and Remedies, D.A.K.

Detlev Parow at World Orphan Drug Congress USA 2019

Linn Parrish, Head Of Corporate Responsibility, Shire

Linn Parrish at World Orphan Drug Congress USA 2019

Gina Parziale, Executive Director, Alport Syndrome Foundation

Gina Parziale at World Orphan Drug Congress USA 2019

Doug Pfaff, Senior Director, Huron Life Sciences

Doug Pfaff at World Orphan Drug Congress USA 2019

Han Phan, Founder and Executive Officer, Rare Disease Research

Han Phan at World Orphan Drug Congress USA 2019

Anthony Philippakis, Chief Data Officer, Broad Institute

Anthony Philippakis at World Orphan Drug Congress USA 2019

Mathew Pletcher, Head of Rare Disease Discovery, Roche

Mathew Pletcher at World Orphan Drug Congress USA 2019

Mathew T. Pletcher, Head of Rare Disease Discovery, Roche

Mathew T. Pletcher at World Orphan Drug Congress USA 2019

Antonio Porras, Scientist, Children's National Health System

Antonio Porras at World Orphan Drug Congress USA 2019

Tanya Potashnik, Director Policy & Economic Analysis, Patented Medicine Prices Review Board, Goverment Of Canada

Tanya Potashnik at World Orphan Drug Congress USA 2019

Sarah Procario, Advocacy-Communications Manager, The Hemophilia Foundation of Michigan

Sarah Procario at World Orphan Drug Congress USA 2019

Mingxin Qian, Chief Executive Officer, Tongli Biomedical

Mingxin Qian at World Orphan Drug Congress USA 2019

Janet Rae, Vice President Of Global Head Of Regulatory Affairs, Ultragenyx Pharmaceutical

Janet Rae at World Orphan Drug Congress USA 2019

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Harsha Rajasimha at World Orphan Drug Congress USA 2019

Julie Raskin, Executive Director, Congenital Hyperinsulinism International

Julie Raskin at World Orphan Drug Congress USA 2019

Trevor Richter, Director, Common Drug Review and Optimal Use, CADTH

Trevor Richter at World Orphan Drug Congress USA 2019

Kaye Robertson, Assistant Director, Australian Government Department of Health

Kaye Robertson at World Orphan Drug Congress USA 2019

Luke Rosen, Head of Patient Engagement and Digital Health Experience, Ovid Therapeutics

Luke Rosen at World Orphan Drug Congress USA 2019

Theodore Roth, MD/PhD Student, University of California, San Francisco

Theodore Roth at World Orphan Drug Congress USA 2019

Mark Rothera, President and Chief Executive Officer, Orchard Therapeutics

Mark Rothera at World Orphan Drug Congress USA 2019

Peter Saltonstall, President and Chief Executive Officer, National Organization for Rare Disorders

Peter Saltonstall at World Orphan Drug Congress USA 2019

Lori Sames, Founder and President, Hannah's Hope Fund

Lori Sames at World Orphan Drug Congress USA 2019

Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

Scott Schliebner at World Orphan Drug Congress USA 2019

Denise Scots Knight, Chief Executive Officer and Founder, Mereo BioPharma

Denise Scots Knight at World Orphan Drug Congress USA 2019

Samir Shaikh, Deputy Director, F.D.A. U.S. Food and Drug Administration

Samir Shaikh at World Orphan Drug Congress USA 2019

Jeffrey Sherman, Chief Medical Officer, Horizon Pharma

Jeffrey Sherman at World Orphan Drug Congress USA 2019

Kristin Smedley, President, Curing Retinal Blindness Foundation

Kristin Smedley at World Orphan Drug Congress USA 2019

Josh Sommer, Co Founder And Executive Director, Chordoma Foundation

Josh Sommer at World Orphan Drug Congress USA 2019

Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics

Frank Sorgi at World Orphan Drug Congress USA 2019

David Sourdive, Executive Vice President Technical Operations, Cellectis

David Sourdive at World Orphan Drug Congress USA 2019

Marshall Summar, Division Chief of Genetics and Metabolism, Children's National Medical Center

Marshall Summar at World Orphan Drug Congress USA 2019

Jennifer Thornton, Executive Director, A.T. Childrens Project

Jennifer Thornton at World Orphan Drug Congress USA 2019

Barry Ticho, Chief Medical Officer, Stoke Therapeutics

Barry Ticho at World Orphan Drug Congress USA 2019

Julie Tierney, Senior Policy Advisor for Strategic Planning and Legislation, F.D.A. U.S. Food and Drug Administration

Julie Tierney at World Orphan Drug Congress USA 2019

Dan Tierno, Strategic Implementation Manager, Global Data Sciences and Analytics, Bayer

Dan Tierno at World Orphan Drug Congress USA 2019

Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria

Vladimir Tomov at World Orphan Drug Congress USA 2019

Laura Trutoiu, Director of Research, Association For Creatine Deficiencies

Laura Trutoiu at World Orphan Drug Congress USA 2019

Sheela Upadhyaya, Associate Director Highly Specialised Technologies, National Institute for Health and Care Excellence

Sheela Upadhyaya at World Orphan Drug Congress USA 2019

Tiina Urv, Program Director, National Center for Advancing Translational Sciences - NCATS

Tiina Urv at World Orphan Drug Congress USA 2019

Margaret Vernon, Vice President and General Manager, Outcomes Research, Evidera

Margaret Vernon at World Orphan Drug Congress USA 2019

Alain Vicari, Chief Executive Officer and Founder, Calypso Biotech SA

Alain Vicari at World Orphan Drug Congress USA 2019

Monica Weldon, Chief Executive Officer, Bridge the Gap -SYNGAP Educatrion and Research Foundation

Monica Weldon at World Orphan Drug Congress USA 2019

James Wilson, Director of Gene Therapy Program, University of Pennsylvania School Of Medicine

James Wilson at World Orphan Drug Congress USA 2019

Joe Young, Chief Business Officer, Araim Pharmaceuticals Inc

Joe Young at World Orphan Drug Congress USA 2019