2020 Speakers

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

 

Rebecca Abbott, Office of Government Affairs, March of Dimes

Rebecca Abbott | Office of Government Affairs | March of Dimes » speaking at Orphan USA

Derek Adams, Chief Technology And Manufacturing Officer, bluebird bio

Derek Adams | Chief Technology And Manufacturing Officer | bluebird bio » 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

Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation

Kristin Anthony | President | Pten Hamartoma Tumor Syndrome Foundation » 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

Rasappa Arumugham, Chief Scientific Officer, Ocugen

Rasappa Arumugham | Chief Scientific Officer | Ocugen » 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

Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH)

Christopher Austin | Director, National Center For Advancing Translational Sciences (Ncats) | National Institutes of Health (NIH) » speaking at Orphan USA

Lakiea Bailey, Executive Director, Sickle Cell Consortium

Lakiea Bailey | Executive Director | Sickle Cell Consortium » speaking at Orphan USA

Annette Bakker, President, Childrens Tumor Foundation

Annette Bakker | President | Childrens Tumor Foundation » 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

Shannon Baumann, Senior Director, Access Value And Evidence Strategy, bluebird bio

Shannon Baumann | Senior Director, Access Value And Evidence Strategy | bluebird bio » speaking at Orphan USA

Allyson Berent, Chief Operating Officer, GeneTx Biotherapeutics, Chief Science Officer,, Foundation for Angelman Syndrome Therapeutics

Allyson Berent | Chief Operating Officer, GeneTx Biotherapeutics, Chief Science Officer, | Foundation for Angelman Syndrome Therapeutics » speaking at Orphan USA

Michelle Berg, President, GMP Nucleic Acids, Aldevron

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

Diane Berry, Vice President, Global Health Policy And Government Affair, Sarepta Therapeutics

Diane Berry | Vice President, Global Health Policy And Government Affair | Sarepta Therapeutics » speaking at Orphan USA

Luc Boileau, Chairman And Chief Executive Officer, INESSS

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

Vanessa Boulanger, Director Of Research Programs, NORD

Vanessa Boulanger | Director Of Research Programs | NORD » speaking at Orphan USA

Paula Boultbee, Chief Commercial Officer, Oncopeptides

Paula Boultbee | Chief Commercial Officer | Oncopeptides » 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

Michael Bozik, President And Chief Executive Officer, Knopp Biosciences

Michael Bozik | President And Chief Executive Officer | Knopp Biosciences » 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

Wilson Bryan, Director, Office of Tissues and Advanced Therapies, CBER, FDA

Wilson Bryan | Director, Office of Tissues and Advanced Therapies, CBER | FDA » 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

Chelsea Catsburg, Engagement Manager, BluePrint Orphan

Chelsea Catsburg | Engagement Manager | BluePrint Orphan » speaking at Orphan USA

Rick Chapman, Director Of Health Economics, ICER

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

Lynsey Chediak, Project Lead, Precision Medicine, World Economic Forum

Lynsey Chediak | Project Lead, Precision Medicine | World Economic Forum » speaking at Orphan USA

Alexander Cole, Executive Director, Global Head Epidemiology, Alexion Pharmaceuticals

Alexander Cole | Executive Director, Global Head Epidemiology | Alexion Pharmaceuticals » speaking at Orphan USA

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors

Ellen Coleman | President And Chief Executive Officer | VOZ Advisors » speaking at Orphan USA

Brett Cook, Senior Associate, F Prime Capital

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

Kathleen Coolidge, Patient Advocacy Manager, Backpack Health

Kathleen Coolidge | Patient Advocacy Manager | Backpack Health » 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

Olivier Danos, Chief Scientific Officer, Regenxbio Inc

Olivier Danos | Chief Scientific Officer | Regenxbio Inc » speaking at Orphan USA

Santanu Das, Managing Director, Huron Consulting Group

Santanu Das | Managing Director | Huron Consulting Group » speaking at Orphan USA

Khrystal K Davis, President & Founder, Texas Rare Alliance

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

Tom Defay, Senior Director, Research And Development, Strategy And Alliance Management, Alexion Pharmaceuticals

Tom Defay | Senior Director, Research And Development, Strategy And Alliance Management | Alexion Pharmaceuticals » speaking at Orphan USA

Ebrahim S Delpassand, Chairman And Chief Executive Officer, RadioMedix Inc.

Ebrahim S Delpassand | Chairman And Chief Executive Officer | RadioMedix Inc. » speaking at Orphan USA

Scott Dorfman, Chief Executive Officer, Odylia Therapeutics

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

Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)

Debbie Drell | Director Of Membership | National Organization for Rare Disorders (NORD) » speaking at Orphan USA

Andrew Ecob, Vice President Of Real World Evidence Strategy, PAREXEL

Andrew Ecob | Vice President Of Real World Evidence Strategy | PAREXEL » speaking at Orphan USA

Rafael Escandon, Vice President Of Development Operations, BridgeBio

Rafael Escandon | Vice President Of Development Operations | BridgeBio » speaking at Orphan USA

Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc

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

Emily Fields, Sr. Manager, Patient Advocacy, bluebird bio

Emily Fields | Sr. Manager, Patient Advocacy | bluebird bio » speaking at Orphan USA

Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge

Amanda Finlayson | Therapeutic Strategy Lead | ClinEdge » speaking at Orphan USA

Kelly Folkers, Senior Research Associate, Division of Medical Ethics,, NYU Langone Medical Center

Kelly Folkers | Senior Research Associate, Division of Medical Ethics, | NYU Langone Medical Center » speaking at Orphan USA

Mani Foroohar, Managing Director, Genetic Medicines, SVB Leerink

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

Romina Foster-Bonds, Programs Director, CureDuchenne

Romina Foster-Bonds | Programs Director | CureDuchenne » 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

Pat Furlong, Founding President And Chief Executive Officer, Parent Project Muscular Dystrophy

Pat Furlong | Founding President And Chief Executive Officer | Parent Project Muscular Dystrophy » speaking at Orphan USA

Kamilla Gaitova, Head of Center for Health Economics and Health Technology Assessment, Republican Center for Health Development, Kazakhstan

Kamilla Gaitova | Head of Center for Health Economics and Health Technology Assessment | 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

Barbara Goodman, President And Chief Operating Officer, Cures Within Reach

Barbara Goodman | President And Chief Operating Officer | Cures Within Reach » speaking at Orphan USA

Amy Gray, Chief Executive Officer, Charcot-Marie-Tooth Association

Amy Gray | Chief Executive Officer | Charcot-Marie-Tooth Association » 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

Mike Hale, Vice President and Chief Statistical Scientist, Takeda Pharmaceuticals

Mike Hale | Vice President and Chief Statistical Scientist | Takeda Pharmaceuticals » speaking at Orphan USA

Jennifer Helfer, Director, Global Patient Advocacy Lead, bluebird bio

Jennifer Helfer | Director, Global Patient Advocacy Lead | bluebird bio » speaking at Orphan USA

Andrew Hobbs, Managing Director, Huron Consulting Group

Andrew Hobbs | Managing Director | Huron Consulting Group » speaking at Orphan USA

Bruce Hochstadt, Chief Operating Officer, Grace Therapeutics

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

Melissa Hogan, Founder And President, Project Alive

Melissa Hogan | Founder And President | Project Alive » speaking at Orphan USA

Kui Huang, Rare Disease Epidemiology Lead Strategist, Pfizer

Kui Huang | Rare Disease Epidemiology Lead Strategist | Pfizer » 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

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

Emil Kakkis | Chief Executive Officer | Ultragenyx Pharmaceutical » speaking at Orphan USA

Sujay Kango, Chief Commercial Officer, Acceleron Pharma

Sujay Kango | Chief Commercial Officer | Acceleron Pharma » speaking at Orphan USA

Rashida Karmali, Chief Executive Officer, Tactical Therapeutics Inc

Rashida Karmali | Chief Executive Officer | Tactical Therapeutics Inc » speaking at Orphan USA

Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Therapeutics

Vikram Karnani | Executive Vice President, Chief Commercial Officer | Horizon Therapeutics » 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

Maya Khurana, Manager, Huron Consulting Group

Maya Khurana | Manager | Huron Consulting Group » speaking at Orphan USA

Prashant Kohli, Vice President Commercial Operations, Grace Therapeutics

Prashant Kohli | Vice President Commercial Operations | Grace Therapeutics » speaking at Orphan USA

Jonathan Kornstein, Executive Director, Program Strategy, Premier Research

Jonathan Kornstein | Executive Director, Program Strategy | Premier Research » speaking at Orphan USA

Mark Kroes, CEO, Global Data Support

Mark Kroes | CEO | Global Data Support » speaking at Orphan USA

Salvatore La Rosa, Chief Scientific Officer, Children's Tumor Foundation

Salvatore La Rosa | Chief Scientific Officer | Children's Tumor Foundation » speaking at Orphan USA

Steven Laffoon, President, Wylder Nation

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

Tom Lambdin, Staff, Ben's Friends

Tom Lambdin | Staff | Ben's Friends » speaking at Orphan USA

Janet Lynch Lambert, Chief Executive Officer, Alliance for Regenerative Medicine

Janet Lynch Lambert | Chief Executive Officer | Alliance for Regenerative Medicine » speaking at Orphan USA

Jim Lang, Chief Executive Officer, EVERSANA

Jim Lang | Chief Executive Officer | EVERSANA » speaking at Orphan USA

David Lapidus, President, Lapidus Data

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

Kimberly Leblanc, Associate Director Of Research Operations Undiagnosed Diseases Network Coordinating Center, Harvard Medical School

Kimberly Leblanc | Associate Director Of Research Operations Undiagnosed Diseases Network Coordinating Center | Harvard Medical School » speaking at Orphan USA

Michael Lehmicke, Director, Science And Industry Affairs, Alliance for Regenerative Medicine

Michael Lehmicke | Director, Science And Industry Affairs | Alliance for Regenerative Medicine » speaking at Orphan USA

Clement Leong, Chief Executive Officer and Managing Director, Stamford Pharmaceuticals

Clement Leong | Chief Executive Officer and Managing Director | Stamford Pharmaceuticals » 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

Donald Lo, Director, Therapeutic Development Branch, Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences - NCATS

Donald Lo | Director, Therapeutic Development Branch, Division of Pre-Clinical Innovation | National Center for Advancing Translational Sciences - NCATS » speaking at Orphan USA

Robert Long, Executive Director Of Strategic Development, Uplifting Athletes

Robert Long | Executive Director Of Strategic Development | Uplifting Athletes » 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

Jenn Mcnary, Consultant, Duchenne Parent Advocate

Jenn Mcnary | Consultant | Duchenne Parent Advocate » 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

Sheila Mikhail, President and Co-Founder, Asklepios BioPharmaceutical Inc

Sheila Mikhail | President and Co-Founder | Asklepios BioPharmaceutical Inc » speaking at Orphan USA

Timothy Miller, Co-Founder, President and Chief Scientific Officer, Abeona Therapeutics

Timothy Miller | Co-Founder, President and Chief Scientific Officer | Abeona Therapeutics » speaking at Orphan USA

Debra Miller, Founder And President, CureDuchenne

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

James Miskin, Chief Technical Officer, Oxford Biomedica

James Miskin | Chief Technical Officer | Oxford Biomedica » speaking at Orphan USA

Samarendra Mohanty, Founder And President, NanoScope Technologies

Samarendra Mohanty | Founder And President | NanoScope Technologies » 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

Lauren Neighbours, Neighbours, Senior Director, Regulatory Affairs, Optum Digital Research Network, Optum

Lauren Neighbours | Neighbours, Senior Director, Regulatory Affairs, Optum Digital Research Network | Optum » 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

Sara Nochur, Chief Regulatory Officer, Regulatory Affairs, Alnylam Pharaceuticals

Sara Nochur | Chief Regulatory Officer, Regulatory Affairs | Alnylam Pharaceuticals » speaking at Orphan USA

Daniel O'Mahony, Partner, Seroba Life Sciences

Daniel O'Mahony | Partner | Seroba Life Sciences » speaking at Orphan USA

Nawal Ouzren, Chief Executive Officer, Sensorion Pharma

Nawal Ouzren | Chief Executive Officer | Sensorion Pharma » speaking at Orphan USA

Arrigo Paciello, Scientific Advisor, AIFA

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

Nick Page, Chief Operations Officer, Oxford Biomedica

Nick Page | Chief Operations Officer | Oxford Biomedica » speaking at Orphan USA

Palani Palaniappan, SVP, Head of Global Technical Operations, Sarepta Therapeutics

Palani Palaniappan | SVP, Head of Global Technical Operations | Sarepta Therapeutics » speaking at Orphan USA

Jim Palma, Executive Director, TargetCancer Foundation

Jim Palma | Executive Director | TargetCancer Foundation » speaking at Orphan USA

Francis Pang, Vice-President Global Market Access, Orchard Therapeutics

Francis Pang | Vice-President Global Market Access | Orchard Therapeutics » 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

Linn Parrish, Head of CSR Innovation and Stakeholder Engagement, Corporate Communications & Public Affairs, Takeda

Linn Parrish | Head of CSR Innovation and Stakeholder Engagement, Corporate Communications & Public Affairs | Takeda » speaking at Orphan USA

Doug Paul, Vice President And Partner, MME

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

Steven Peskin, Associate Clinical Professor Of Medicine, Rutgers Rwj Medical School, Executive Medical Director Population Health And Transform, Horizon Blue Cross Blue Shield - New Jersey

Steven Peskin | Associate Clinical Professor Of Medicine, Rutgers Rwj Medical School, Executive Medical Director Population Health And Transform | Horizon Blue Cross Blue Shield - New Jersey » 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

Mayo Pujols, Vice President, Head Of Global Cell, Gene Technical Development And Manufacturing, Novartis

Mayo Pujols | Vice President, Head Of Global Cell, Gene Technical Development And Manufacturing | Novartis » 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

Karlyne Reilly, Senior Associate Scientist And Section Head Of, National Cancer Institute

Karlyne Reilly | Senior Associate Scientist And Section Head Of | National Cancer Institute » speaking at Orphan USA

John Reynders, Vice President, Data Sciences, Genomics, and Bioinformatics, Alexion Pharmaceticals

John Reynders | Vice President, Data Sciences, Genomics, and Bioinformatics | Alexion Pharmaceticals » speaking at Orphan USA

Marisa Ricci, Vice President Patient Diagnosis Programs & U.S. Field Medical Engagement, Ultragenyx Pharmaceutical

Marisa Ricci | Vice President Patient Diagnosis Programs & U.S. Field Medical Engagement | Ultragenyx Pharmaceutical » 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

Seth Rotberg, Co Founder and President, Our Odyssey

Seth Rotberg | Co Founder and President | Our Odyssey » speaking at Orphan USA

John Rother, Chief Executive Officer, National Coalition On Health Care

John Rother | Chief Executive Officer | National Coalition On Health Care » speaking at Orphan USA

Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics

Mark Rothera | President And Chief Executive Officer | Orchard 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

Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)

Peter Saltonstall | Chief Executive Officer | National Organization for Rare Disorders (NORD) » speaking at Orphan USA

Abby Sandler, Executive Director, MyPART, Center for Cancer Research,, National Institutes of Health

Abby Sandler | Executive Director, MyPART, Center for Cancer Research, | National Institutes of Health » speaking at Orphan USA

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

Scott Schliebner | Vice President, Scientific Affairs, Rare Diseases | PRA Health Sciences » speaking at Orphan USA

Sophie Schmitz, Managing Partner, Partners4Access

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

Jeffrey Schwartz, Managing Director, Bain Capital Life Sciences

Jeffrey Schwartz | Managing Director | Bain Capital Life Sciences » speaking at Orphan USA

Jim Shaffer, Cbo, Eiger Biopharmaceuticals

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

Lesha Shah, Co-Chair, Pediatric Gene Therapy & Medical Ethics Working Group, Assistant Professor of Psychiatry, Icahn School of Medicine, The Mount Sinai Hospital

Lesha Shah | Co-Chair, Pediatric Gene Therapy & Medical Ethics Working Group, Assistant Professor of Psychiatry, Icahn School of Medicine | The Mount Sinai Hospital » speaking at Orphan USA

Nataliya Shandarovska, Patient Advocate, National Alliance for Rare Diseases Support - Malta

Nataliya Shandarovska | Patient Advocate | National Alliance for Rare Diseases Support - Malta » 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

Gerard Short, Senior Vice President, Clinical And Regulatory, Freeline Therapeutics

Alison Skrinar, Vice President, Clinical Outcomes Research and Evaluation, Ultragenyx Pharmaceutical

Alison Skrinar | Vice President, Clinical Outcomes Research and Evaluation | Ultragenyx Pharmaceutical » 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

Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc

Meagan Spychala | Assistant Vice President Of Patient Engagement And Program Strategy | Rho, Inc » 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

Becky Thompson, Director Integrated Solutions, PAREXEL

Becky Thompson | Director Integrated Solutions | PAREXEL » 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

Brigette Tippin Davis, Senior Vice President, R&D, Ambry Genetics

Brigette Tippin Davis | Senior Vice President, R&D | Ambry Genetics » speaking at Orphan USA

Ngeh Toyang, Chief Executive Officer, Flavocure Biotech

Ngeh Toyang | Chief Executive Officer | Flavocure Biotech » 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

Jodie Vento, Chair, Access Committee, National Society of Genetic Counselors, Manager, Center for Rare Disease Therapy, Childrens Hospital of Pittsburgh

Jodie Vento | Chair, Access Committee, National Society of Genetic Counselors, Manager, Center for Rare Disease Therapy | Childrens Hospital of Pittsburgh » speaking at Orphan USA

Terry Jo Vetters Bichell, Founder and Director, COMBINEDBrain

Terry Jo Vetters Bichell | Founder and Director | COMBINEDBrain » speaking at Orphan USA

Kim Warren, Head Of Operations, Avrobio

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

Melissa Wasserstein, Chief, Division of Pediatric Genetic Medicine, Department of Pediatrics,, Montefiore Medical Center

Melissa Wasserstein | Chief, Division of Pediatric Genetic Medicine, Department of Pediatrics, | Montefiore Medical Center » speaking at Orphan USA

Monica Weldon, CEO, Bridge The Gap Syngap Education And Research Foundation

Monica Weldon | CEO | Bridge The Gap Syngap Education And Research Foundation » speaking at Orphan USA

Terry Wilcox, Co-Founder and Executive Director, Patients Rising & Patients Rising

Terry Wilcox | Co-Founder and Executive Director | Patients Rising & Patients Rising » 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

Janet Woodcock, Director of Drug Evaluation and Research Center, Food and Drug Administration (FDA)

Janet Woodcock | Director of Drug Evaluation and Research Center | Food and Drug Administration (FDA) » speaking at Orphan USA

William Xiang, Director, Strategy and Operations, XW Laboratories

William Xiang | Director, Strategy and Operations | XW Laboratories » speaking at Orphan USA

Richard R Yang, Chief Executive Officer And Founder, Reflection Biotechnologies

Richard R Yang | Chief Executive Officer And Founder | Reflection Biotechnologies » 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

David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.

David Young | Chief Executive Officer | Processa Pharmaceuticals, Inc. » speaking at Orphan USA

Timothy Yu, Attending Physician, Investigator, Division Of Genetics And Genomics, Boston Children's Hospital

Timothy Yu | Attending Physician, Investigator, Division Of Genetics And Genomics | Boston Children's Hospital » speaking at Orphan USA

Ran Zheng, Chief Technical Officer, Orchard Therapeutics

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