2020 Speakers

World Orphan Drug Congress USA will feature 350+ speakers from pharma, biotechs, government, regulators, patient groups & service providers. 

 

Saraswathy (Sara) Nochur, Senior Vice President Regulatory Affairs, Alnylam Pharaceuticals

Saraswathy (Sara) Nochur | Senior Vice President Regulatory Affairs | Alnylam Pharaceuticals » speaking at Orphan USA

Renee Aguiar-Lucander, Chief Executive Officer, Calliditas Therapeutics

Renee Aguiar-Lucander | Chief Executive Officer | Calliditas Therapeutics » speaking at Orphan USA

Jeonghoon Ahn, Director, National Evidence-based Healthcare Collaborating Agency

Jeonghoon Ahn | Director | National Evidence-based Healthcare Collaborating Agency » speaking at Orphan USA

Anjan Aralihalli, Venture Partner, C.T.I. life Sciences Fund

Anjan Aralihalli | Venture Partner | C.T.I. life Sciences Fund » speaking at Orphan USA

Diego Ardigo, R&D Rare Diseases Unit Head, Chiesi Farmaceutici SpA

Diego Ardigo | R&D Rare Diseases Unit Head | Chiesi Farmaceutici SpA » speaking at Orphan USA

Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, National Institute for Health and Disability Insurance RIZIV-INAMI

Francis Arickx | Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy | National Institute for Health and Disability Insurance RIZIV-INAMI » speaking at Orphan USA

Rania Ashraf, Associate Director Market Access, Janssen

Rania Ashraf | Associate Director Market Access | Janssen » speaking at Orphan USA

Kagan Atikeler, Pharmacist, Turkish Medicines and Medical Devices Agency

Kagan Atikeler | Pharmacist | Turkish Medicines and Medical Devices Agency » speaking at Orphan USA

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

Ron Bartek | President, Director, And Founder | Friedreich's Ataxia Research Alliance » speaking at Orphan USA

Michelle Berg, President, GMP Nucleic Acids, Aldevron

Michelle Berg | President, GMP Nucleic Acids | Aldevron » speaking at Orphan USA

Luc Boileau, Chairman And Chief Executive Officer, INESSS

Luc Boileau | Chairman And Chief Executive Officer | INESSS » speaking at Orphan USA

Alfred Boyle, Senior Vice President Technical Operations, Alnylam Pharmaceuticals

Alfred Boyle | Senior Vice President Technical Operations | Alnylam Pharmaceuticals » speaking at Orphan USA

P Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)

P Brooks | Program Director, National Center For Advancing Translational Sciences - Ncats | National Institutes of Health (NIH) » speaking at Orphan USA

Michael Brudno, Scientific Director of Centre for Computational Medicine, Hospital for Sick Children

Michael Brudno | Scientific Director of Centre for Computational Medicine | Hospital for Sick Children » speaking at Orphan USA

Chuck Bucklar, Group VP, Commercial Operations North America, BioMarin Pharmaceutical Inc.

Chuck Bucklar | Group VP, Commercial Operations North America | BioMarin Pharmaceutical Inc. » speaking at Orphan USA

Anna Bucsics, Project Advisor, MoCA - Mechanism of Coordinated Access to Orphan M

Anna Bucsics | Project Advisor | MoCA - Mechanism of Coordinated Access to Orphan M » speaking at Orphan USA

Rick Chapman, Director Of Health Economics, ICER

Rick Chapman | Director Of Health Economics | ICER » speaking at Orphan USA

Brett Cook, Senior Associate, F Prime Capital

Brett Cook | Senior Associate | F Prime Capital » speaking at Orphan USA

Gavin Corcoran, Chief Research And Development Officer, Axovant Sciences

Gavin Corcoran | Chief Research And Development Officer | Axovant Sciences » speaking at Orphan USA

Anne Cropp, Chief Scientific Officer, Early Access Care

Anne Cropp | Chief Scientific Officer | Early Access Care » speaking at Orphan USA

Omar Dabbous, Vice President, Health Economics And Outcomes Research, AveXis

Omar Dabbous | Vice President, Health Economics And Outcomes Research | AveXis » speaking at Orphan USA

Khrystal K Davis, President & Founder, Texas Rare Alliance

Khrystal K Davis | President & Founder | Texas Rare Alliance » speaking at Orphan USA

Scott Dorfman, Chief Executive Officer, Odylia Therapeutics

Scott Dorfman | Chief Executive Officer | Odylia Therapeutics » speaking at Orphan USA

Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc

Vivian Fernandez | Director Of Patient Advocacy | Regenxbio Inc » speaking at Orphan USA

Mani Foroohar, Managing Director, Genetic Medicines, SVB Leerink

Mani Foroohar | Managing Director, Genetic Medicines | SVB Leerink » speaking at Orphan USA

Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)

Andrea Furia-Helms | Director, Patient Affairs Staff | U.S. Food and Drug Administration (FDA) » speaking at Orphan USA

Kamilla Gaitova, Head of HTA Department, Republican Center for Health Development, Kazakhstan

Kamilla Gaitova | Head of HTA Department | Republican Center for Health Development, Kazakhstan » speaking at Orphan USA

Mark Galbraith, Head Of Quality Control And Analytical Sciences, Spark Therapeutics

Mark Galbraith | Head Of Quality Control And Analytical Sciences | Spark Therapeutics » speaking at Orphan USA

Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders (NORD)

Pamela Gavin | Chief Strategy Officer | National Organization for Rare Disorders (NORD) » speaking at Orphan USA

Scott Gray, Chief Executive Officer, Clincierge

Scott Gray | Chief Executive Officer | Clincierge » speaking at Orphan USA

Bernard Grimm, Director, EuropaBio

Bernard Grimm | Director | EuropaBio » speaking at Orphan USA

Bruce Hochstadt, Chief Operating Officer, Grace Therapeutics

Bruce Hochstadt | Chief Operating Officer | Grace Therapeutics » speaking at Orphan USA

Kevin Huang, President, Chinese Organization for Rare Disorders

Kevin Huang | President | Chinese Organization for Rare Disorders » speaking at Orphan USA

Adam Hutchings, Managing Director, Dolon

Adam Hutchings | Managing Director | Dolon » speaking at Orphan USA

Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical

Thomas Kassberg | Chief Business Officer And Executive Vice President | Ultragenyx Pharmaceutical » speaking at Orphan USA

Ken Kengatharan, Managing Partner, Atheneos Ventures And Chief Executive Officer, Atheneos Ventures

Ken Kengatharan | Managing Partner, Atheneos Ventures And Chief Executive Officer | Atheneos Ventures » speaking at Orphan USA

Steven Laffoon, President, Wylder Nation

Steven Laffoon | President | Wylder Nation » speaking at Orphan USA

David Lapidus, President, Lapidus Data

David Lapidus | President | Lapidus Data » speaking at Orphan USA

Friedhelm Leverkus, Director Health Technology Assessment And Outcomes Research, Pfizer

Friedhelm Leverkus | Director Health Technology Assessment And Outcomes Research | Pfizer » speaking at Orphan USA

Edmund Lim, Co-Founder, We CARE Journey

Edmund Lim | Co-Founder | We CARE Journey » speaking at Orphan USA

Marius George Linguraru, Principal Investigator, Children's National Medical Center

Marius George Linguraru | Principal Investigator | Children's National Medical Center » speaking at Orphan USA

Pamela Mace, President, Fibromuscular Dysplasia Society of America

Pamela Mace | President | Fibromuscular Dysplasia Society of America » speaking at Orphan USA

Sandy Macrae, President And Chief Executive Officer, Sangamo Therapeutics

Sandy Macrae | President And Chief Executive Officer | Sangamo Therapeutics » speaking at Orphan USA

Peter Marks, Director, Center For Biologics Evaluation And Research (Cber), U.S. Food and Drug Administration

Peter Marks | Director, Center For Biologics Evaluation And Research (Cber) | U.S. Food and Drug Administration » speaking at Orphan USA

Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Inc.

Paolo Martini | Chief Scientific Officer, Rare Diseases | Moderna Inc. » speaking at Orphan USA

Janet Maynard, Director, Office Of Orphan Products Development, Food And Drug Administration, U.S. Food and Drug Administration (FDA)

Janet Maynard | Director, Office Of Orphan Products Development, Food And Drug Administration | U.S. Food and Drug Administration (FDA) » speaking at Orphan USA

Mary Mcgowan, Executive Director, The Myositis Association

Mary Mcgowan | Executive Director | The Myositis Association » speaking at Orphan USA

Parag Meswani, Senior Vice President Of Commercial Strategy And Operations, Axovant Sciences

Parag Meswani | Senior Vice President Of Commercial Strategy And Operations | Axovant Sciences » speaking at Orphan USA

Debra Miller, Founder And President, CureDuchenne

Debra Miller | Founder And President | CureDuchenne » speaking at Orphan USA

Nuriya Musina, Head Of Development, Federal State Budgetary Institution, the Center for Healthcare Quality Assessme, Ministry of Health Russian Federation

Nuriya Musina | Head Of Development, Federal State Budgetary Institution, the Center for Healthcare Quality Assessme | Ministry of Health Russian Federation » speaking at Orphan USA

Joseph Musumeci, President, BluePrint Orphan

Joseph Musumeci | President | BluePrint Orphan » speaking at Orphan USA

Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

Alexander Natz | Secretary General | European Confederation of Pharmaceutical Entrepreneurs (Belgium) » speaking at Orphan USA

Yukiko Nishimura, President, ASrid Japan

Yukiko Nishimura | President | ASrid Japan » speaking at Orphan USA

Neena Nizar, President, The Jansens Foundation

Neena Nizar | President | The Jansens Foundation » speaking at Orphan USA

Arrigo Paciello, Scientific Advisor, AIFA

Arrigo Paciello | Scientific Advisor | AIFA » speaking at Orphan USA

Art Pappas, Managing Partner, Pappas Capital

Art Pappas | Managing Partner | Pappas Capital » speaking at Orphan USA

Anne Pariser, Director, Office Of Rare Diseases Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)

Anne Pariser | Director, Office Of Rare Diseases Research, National Center For Advancing Translational Sciences - Ncats | National Institutes of Health (NIH) » speaking at Orphan USA

Detlev Parow, Head Pharmaceutical Department, D.A.K.

Detlev Parow | Head Pharmaceutical Department | D.A.K. » speaking at Orphan USA

Doug Paul, Vice President And Partner, MME

Doug Paul | Vice President And Partner | MME » speaking at Orphan USA

Andres Pichon-Riviere, Executive Director of the Institute for Clinical Effectiveness and Health Policy (IECS), Director of the Health Technology, University Of Buenos Aires

Andres Pichon-Riviere | Executive Director of the Institute for Clinical Effectiveness and Health Policy (IECS), Director of  the Health Technology | University Of Buenos Aires » speaking at Orphan USA

Oresta Piniazhko, Director, HTA Department, Ministry of Health of Ukraine

Oresta Piniazhko | Director, HTA Department | Ministry of Health of Ukraine » speaking at Orphan USA

Nader Z Pourhassan, President And Chief Executive Officer, CytoDyn, Inc.

Nader Z Pourhassan | President And Chief Executive Officer | CytoDyn, Inc. » speaking at Orphan USA

Harsha Rajasimha, Co Founder, Organization for Rare Diseases India

Harsha Rajasimha | Co Founder | Organization for Rare Diseases India » speaking at Orphan USA

Shankar Ramaswamy, Chief Business Officer, Axovant

Shankar Ramaswamy | Chief Business Officer | Axovant » speaking at Orphan USA

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

Trevor Richter | Director, Common Drug Review And Optimal Use | CADTH » speaking at Orphan USA

Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries

Shawn Ritchie | Chief Executive Officer | Med-Life Discoveries » speaking at Orphan USA

Juan Roman, Head Of Market Access And Pricing, Acceleron Pharmaceuticals, Inc.

Juan Roman | Head Of Market Access And Pricing | Acceleron Pharmaceuticals, Inc. » speaking at Orphan USA

Luke Rosen, Head Of Patient Engagement And Digital Health Experience, Ovid Therapeutics

Luke Rosen | Head Of Patient Engagement And Digital Health Experience | Ovid Therapeutics » speaking at Orphan USA

Delphine Roulland, Director Government Affairs And Public Policy, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

Delphine Roulland | Director Government Affairs And Public Policy | European Confederation of Pharmaceutical Entrepreneurs (Belgium) » speaking at Orphan USA

Sophie Schmitz, Managing Partner, Partners4Access

Sophie Schmitz | Managing Partner | Partners4Access » speaking at Orphan USA

Jim Shaffer, Cbo, Eiger Biopharmaceuticals

Jim Shaffer | Cbo | Eiger Biopharmaceuticals » speaking at Orphan USA

Panna Sharma, President and Chief Executive Officer, Lantern Pharma

Panna Sharma | President and Chief Executive Officer | Lantern Pharma » speaking at Orphan USA

Rachel Sher, Vice President Policy And Regulatory Affairs, NORD

Rachel Sher | Vice President Policy And Regulatory Affairs | NORD » speaking at Orphan USA

Alex Sherman, Director, Center For Innovation And Bioinformatics, Harvard Medical School

Alex Sherman | Director, Center For Innovation And Bioinformatics | Harvard Medical School » speaking at Orphan USA

Kristin Smedley, President, Curing Retinal Blindness Foundation

Kristin Smedley | President | Curing Retinal Blindness Foundation » speaking at Orphan USA

Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer

Bob Smith | Senior Vice President, Global Gene Therapy Business | Pfizer » speaking at Orphan USA

Marshall Summar, Division Chief Of Genetics And Metabolism, Children's National Medical Center

Marshall Summar | Division Chief Of Genetics And Metabolism | Children's National Medical Center » speaking at Orphan USA

Weiyong Sun, Senior Director, Specialty Medicine Search, Evaluation And Global Business Development, Daiichi Sankyo Co Ltd

Weiyong Sun | Senior Director, Specialty Medicine Search, Evaluation And Global Business Development | Daiichi Sankyo Co Ltd » speaking at Orphan USA

Thayer Surette, Deputy Director, Partnership to Improve Patient Care (PIPC)

Thayer Surette | Deputy Director | Partnership to Improve Patient Care (PIPC) » speaking at Orphan USA

Stephanie Tagliatela, Co-Founder And Chief Scientific Officer, Encoded Therapeutics

Stephanie Tagliatela | Co-Founder And Chief Scientific Officer | Encoded Therapeutics » speaking at Orphan USA

Dan Tierno, Strategic Implementation Manager, Global Data Sciences And Analytics, Bayer US LLC

Dan Tierno | Strategic Implementation Manager, Global Data Sciences And Analytics | Bayer US LLC » speaking at Orphan USA

Sara Traigle Van Geertruyden, Executive Director, Partnership to Improve Patient Care (PIPC)

Sara Traigle Van Geertruyden | Executive Director | Partnership to Improve Patient Care (PIPC) » speaking at Orphan USA

Evert Jan Van Lente, Director Of Eu Affairs, AOK Bundesverdand

Evert Jan Van Lente | Director Of Eu Affairs | AOK Bundesverdand » speaking at Orphan USA

Carole Spangler Vaughn, Chief Executive Officer, Emtora Biosciences

Carole Spangler Vaughn | Chief Executive Officer | Emtora Biosciences » speaking at Orphan USA

Kim Warren, Head Of Operations, Avrobio

Kim Warren | Head Of Operations | Avrobio » speaking at Orphan USA

Monica Weldon, President and Chief Executive Officer, Bridge The Gap Syngap Education And Research Foundation

Monica Weldon | President and Chief Executive Officer | Bridge The Gap Syngap Education And Research Foundation » speaking at Orphan USA

Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders

Durhane Wong-Rieger | President And Chief Executive Officer | Canadian Organization For Rare Disorders » speaking at Orphan USA

Dimitar Yonchev, Patient Advocate Representative, Pro Retina Deutschland e.V.

Dimitar Yonchev | Patient Advocate Representative | Pro Retina Deutschland e.V. » speaking at Orphan USA

Ran Zheng, Chief Technical Officer, Orchard Therapeutics

Ran Zheng | Chief Technical Officer | Orchard Therapeutics » speaking at Orphan USA