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 Foundation

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

Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma

Thomas Abbott | Head, Real World Data And Evidence | Astellas Pharma » 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

Sherilyn Adcock, Executive Vice President, Medical And Scientific Affairs, Worldwide Clinical Trials

Sherilyn Adcock | Executive Vice President, Medical And Scientific Affairs | Worldwide Clinical Trials » speaking at Orphan USA

Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals

Sonalee Agarwal | Vice President Of Value And Evidence Strategy | Alnylam Pharmaceuticals » 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

Sean Ainsworth, Chief Executive Officer And Chairman, Immusoft Corporation

Sean Ainsworth | Chief Executive Officer And Chairman | Immusoft Corporation » speaking at Orphan USA

Derek Ansel, Director, Rare, Orphan and Pediatric Diseases, Synteract

Derek Ansel | Director, Rare, Orphan and Pediatric Diseases | Synteract » 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, NIHDI - INAMI Belgium

Francis Arickx | Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy | NIHDI - INAMI Belgium » 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

Aisha Asra, Associate Consultant, Huron Consulting Group

Aisha Asra | Associate Consultant | Huron Consulting Group » speaking at Orphan USA

Kagan Atikeler, Researcher, Utrecht University

Kagan Atikeler | Researcher | Utrecht University » 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

Mark Baglin, Vice President Of Global Marketing, Alnylam Pharmaceuticals

Mark Baglin | Vice President Of Global Marketing | Alnylam Pharmaceuticals » 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

Peter Bauer, Chief Genomic Officer, CENTOGENE US, LLC

Peter Bauer | Chief Genomic Officer | CENTOGENE US, LLC » 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

Anny Bedard, Head Of International Business, Commercial Operations, Sarepta Therapeutics

Anny Bedard | Head Of International Business, Commercial Operations | Sarepta Therapeutics » speaking at Orphan USA

Gregory Bell, Group Vice President And Life Sciences Practice Leader, Charles River Associates

Gregory Bell | Group Vice President And Life Sciences Practice Leader | Charles River Associates » speaking at Orphan USA

Alon Ben-Noon, CEO, NeuroSense Therapeutics

Alon Ben-Noon | CEO | NeuroSense Therapeutics » 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 AB

Paula Boultbee | Chief Commercial Officer | Oncopeptides AB » 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

Todd Brady, Chief Executive Officer, Aldeyra Therapeutics

Todd Brady | Chief Executive Officer | Aldeyra Therapeutics » speaking at Orphan USA

Jennifer Bright, Executive Director, Innovation And Value Initiative

Jennifer Bright | Executive Director | Innovation And Value Initiative » speaking at Orphan USA

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

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

Bert Bruce, Vice President, Rare Disease Commercial Development, Pfizer

Bert Bruce | Vice President, Rare Disease Commercial Development | Pfizer » 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 Vice President, Commercial Operations North America, BioMarin Pharmaceutical Inc.

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

Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)

Anna Bucsics | Project Advisor | Mechanism of Coordinated Access to orphan medicinal products (MoCA) » speaking at Orphan USA

Catherine Cancian, VP Pharmaceutical Operations, GenSight Biologics

Catherine Cancian | VP Pharmaceutical Operations | GenSight Biologics » speaking at Orphan USA

Casey Case, Senior Vice President Of Research And Development, 4D Molecular Therapeutics

Casey Case | Senior Vice President Of Research And Development | 4D Molecular Therapeutics » speaking at Orphan USA

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

Alan Cash | Chief Executive Officer And Co-Founder | MetVital » 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

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

Alexander Cole, Executive Director, Global Head Epidemiology, Alexion

Alexander Cole | Executive Director, Global Head Epidemiology | Alexion » 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 Partners

Brett Cook | Senior Associate | F-Prime Capital Partners » 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

Christina Coughlin, Chief Medical Officer, Rubius Therapeutics

Christina Coughlin | Chief Medical Officer | Rubius Therapeutics » speaking at Orphan USA

Benoit Coulombe, Founder, Professor and Director Of Translational Proteomics At The Montreal Clinical Research Institute, Open for Rare

Benoit Coulombe | Founder, Professor and Director Of Translational Proteomics At The Montreal Clinical Research Institute | Open for Rare » speaking at Orphan USA

Anne Cropp, Chief Scientific Officer, Early Access Care

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

John Crowley, Chief Executive Officer, Amicus Therapeutics Inc

John Crowley | Chief Executive Officer | Amicus Therapeutics Inc » speaking at Orphan USA

Pam Crowley Andrews, Co-Founder And Executive Director, Firefly Fund

Pam Crowley Andrews | Co-Founder And Executive Director | Firefly Fund » speaking at Orphan USA

Daniel Curran, Head, Rare Diseases Therapeutic Area Unit, Takeda

Daniel Curran | Head, Rare Diseases Therapeutic Area Unit | Takeda » 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

Yong Dai, Chief Technology Officer, Prevail Therapeutics

Yong Dai | Chief Technology Officer | Prevail Therapeutics » 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, Texas Rare Alliance

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

Stratos Davlos, CTO & SVP Managing Director, Innoplexus

Stratos Davlos | CTO & SVP Managing Director | Innoplexus » 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

Eelko Den Breejen, Associate Director, Market Access, Biogen

Eelko Den Breejen | Associate Director, Market Access | Biogen » speaking at Orphan USA

Sheila Diamond, Product Marketing Manager, Medidata, a Dassault Systèmes company

Sheila Diamond | Product Marketing Manager | Medidata, a Dassault Systèmes company » 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

Sean Ekins, Founder And Chief Executive Officer, Collaborations Pharmaceuticals

Sean Ekins | Founder And Chief Executive Officer | Collaborations Pharmaceuticals » speaking at Orphan USA

Rafael Escandon, Vice President Of Development Operations, BridgeBio-Gene Therapy

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

Mark Farmery, Vice President Of Business Development, Neurovive

Mark Farmery | Vice President Of Business Development | Neurovive » speaking at Orphan USA

Lisa Feng, Senior Director, Policy And Advocacy, Cystic Fibrosis Foundation

Lisa Feng | Senior Director, Policy And Advocacy | Cystic Fibrosis Foundation » 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, Senior Manager, Patient Advocacy, bluebird bio

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

Alison Finger, Chief Commercial Officer, bluebird bio

Alison Finger | Chief Commercial Officer | bluebird bio » speaking at Orphan USA

Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge

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

Nasha Fitter, Chief Executive Officer And Head Of Research, FOXG1 Research Foundation

Nasha Fitter | Chief Executive Officer And Head Of Research | FOXG1 Research Foundation » 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

Chad Forinash, Senior Director, Pharmacy And Clinical Services, PharmaCord

Chad Forinash | Senior Director, Pharmacy And Clinical Services | PharmaCord » speaking at Orphan USA

Romina Foster-Bonds, Programs Director, CureDuchenne

Romina Foster-Bonds | Programs Director | CureDuchenne » speaking at Orphan USA

Danielle Friend, Director Of Science And Regulatory Affairs, Biotechnology Industry Organization

Danielle Friend | Director Of Science And Regulatory Affairs | Biotechnology Industry Organization » speaking at Orphan USA

Jon Fryzek, Principal Epidemiologist, Practice Director, Epidemiology, EpidStrategies, A Division of ToxStrategies

Jon Fryzek | Principal Epidemiologist, Practice Director, Epidemiology | EpidStrategies, A Division of ToxStrategies » speaking at Orphan USA

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

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

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

Andrea Furia-Helms | Director, Patient Affairs Staff | U.S. Food and Drug Administration » 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

Bobby Gaspar, Chief Executive Officer, Orchard Therapeutics

Bobby Gaspar | Chief Executive Officer | Orchard 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

Beverly Ginsbury, Managing Director, Huron Consulting Group

Beverly Ginsbury | Managing Director | Huron Consulting Group » 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

Joana Granado, External Innovation Director, Actelion - A Janssen Pharmaceutical Company of Johnson&Johnson

Joana Granado | External Innovation Director | Actelion - A Janssen Pharmaceutical Company of Johnson&Johnson » 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, CEO, Clincierge

Scott Gray | CEO | 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

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

Marianne Hamilton Lopez | Research Director | Duke Margolis Center for Health Policy » speaking at Orphan USA

Michael Hanley, Chief Commercial Officer, Aeglea Biotherapeutics

Michael Hanley | Chief Commercial Officer | Aeglea Biotherapeutics » speaking at Orphan USA

Patrick Haslett, Executive Director Of Clinical Research And Biology, Alnylam Pharmaceuticals

Patrick Haslett | Executive Director Of Clinical Research And Biology | Alnylam Pharmaceuticals » speaking at Orphan USA

Robert Haynes, Senior Director, Global Product And Market Planning, Regenxbio Inc

Robert Haynes | Senior Director, Global Product And Market Planning | Regenxbio Inc » 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

Rachele Hendricks-Sturrup, Health Policy Lead, Future of Privacy Forum

Rachele Hendricks-Sturrup | Health Policy Lead | Future of Privacy Forum » speaking at Orphan USA

Arthur Herman, Fellow, World Economic Forum, Precision Medicine Portfolio, Principal Strategic Consultant, Kaiser Permanente

Arthur Herman | Fellow, World Economic Forum, Precision Medicine Portfolio, Principal Strategic Consultant | Kaiser Permanente » speaking at Orphan USA

Andrew Hobbs, Managing Director, Huron Consulting Group

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

Melissa Hogan, Rare Disease Parent/Advocate, CEO, Doulots

Melissa Hogan | Rare Disease Parent/Advocate, CEO | Doulots » speaking at Orphan USA

Andy Holt, Vice President Of Business Development and Manufacturing, Asklepios BioPharmaceutical Inc

Andy Holt | Vice President Of Business Development and Manufacturing | Asklepios BioPharmaceutical Inc » 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, Director, Dolon

Adam Hutchings | Director | Dolon » speaking at Orphan USA

Gavin Jones, Director of Rare Disease, OPEN Health

Gavin Jones | Director of Rare Disease | OPEN Health » 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, CEO, Tactical Therapeutics Inc

Rashida Karmali | CEO | 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

Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases

Annie Kennedy | Chief of Policy and Advocacy | EveryLife Foundation for Rare Diseases » speaking at Orphan USA

Uday Khire, Chief Executive Officer, AlloMek

Uday Khire | Chief Executive Officer | AlloMek » speaking at Orphan USA

Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson

Sean Khozin | Global Head Of Data Strategy | Johnson & Johnson » speaking at Orphan USA

Maya Khurana, Director, Huron Consulting Group

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

Therese Kinsella, Founder And Chief Scientific Officer, ATXA Therapeutics

Therese Kinsella | Founder And Chief Scientific Officer | ATXA Therapeutics » speaking at Orphan USA

Daniel Knecht, Vice President Of Health Strategy And Innovation, CVS Health

Daniel Knecht | Vice President Of Health Strategy And Innovation | CVS Health » speaking at Orphan USA

Jonathan Kornstein, Executive Director, Program Strategy, Premier Research

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

George Kottayil, Co-Founder And Chief Executive Officer, Grace Therapeutics

George Kottayil | Co-Founder And Chief Executive Officer | Grace Therapeutics » speaking at Orphan USA

Mark Kroes, Chief Executive Officer, Global Data Support

Mark Kroes | Chief Executive Officer | Global Data Support » speaking at Orphan USA

Akshay Kumar, Partner, Partners4Access

Akshay Kumar | Partner | Partners4Access » 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, Executive Director, Ben's Friends

Tom Lambdin | Executive Director | 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

Janice Lansita, Principal Scientist, Practice Director, Biopharmacueticals/Pharmaceuticals, EpidStrategies, A Division of ToxStrategies

Janice Lansita | Principal Scientist, Practice Director, Biopharmacueticals/Pharmaceuticals | EpidStrategies, A Division of ToxStrategies » speaking at Orphan USA

David Lapidus, President, Lapidus Data

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

Yann Le Cam, Chief Executive Officer, EURORDIS

Yann Le Cam | Chief Executive Officer | EURORDIS » 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

Joseph Lee, Executive Director, Analytical Development - Gene Therapy, PTC Therapeutics

Joseph Lee | Executive Director, Analytical Development - Gene Therapy | PTC Therapeutics » 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

Paul Levesque, Global President, Rare Disease, Pfizer

Paul Levesque | Global President, Rare Disease | Pfizer » speaking at Orphan USA

Jeremy Levin, Chairman Of The Board, Directors And Chief Executive Officer, Ovid Therapeutics

Jeremy Levin | Chairman Of The Board, Directors And Chief Executive Officer | Ovid Therapeutics » speaking at Orphan USA

Edmund Lim, Abc, We CARE Journey

Edmund Lim | Abc | We CARE Journey » speaking at Orphan USA

Marius George Linguraru, Principal Investigator at Children’s National Health System, Professor of Radiology, Pediatrics and Biomedical Engineering, The George Washington University

Marius George Linguraru | Principal Investigator at Children’s National Health System, Professor of Radiology, Pediatrics and Biomedical Engineering | The George Washington University » 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 Lodder, President, Biospherics

Robert Lodder | President | Biospherics » speaking at Orphan USA

Brian Loew, Chief Executive Officer, Inspire

Brian Loew | Chief Executive Officer | Inspire » speaking at Orphan USA

Robert Long, Executive Director, Uplifting Athletes

Robert Long | Executive Director | Uplifting Athletes » speaking at Orphan USA

Jarrod Longcor, Chief Business Officer, Cellectar Biosciences Inc

Jarrod Longcor | Chief Business Officer | Cellectar Biosciences Inc » 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

Matthew Majewski, Vice President, Charles River Associates

Matthew Majewski | Vice President | Charles River Associates » 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

Sebastien Martel, Global Head of Rare Diseases, Sanofi

Sebastien Martel | Global Head of Rare Diseases | Sanofi » speaking at Orphan USA

Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Therapeutics

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

Allison May Rosen, Representative, Firefly Fund

Allison May Rosen | Representative | Firefly Fund » speaking at Orphan USA

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

Janet Maynard | Director, Office Of Orphan Products Development, Food And Drug Administration | U.S. Food and Drug Administration » 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

Eileen Mcneill, Programme Manager - Disease Foundations, Oxford University Structural Genomics Consortium

Eileen Mcneill | Programme Manager - Disease Foundations | Oxford University Structural Genomics Consortium » speaking at Orphan USA

Tracy Meffen, VP, Quality & Regulatory Affairs,, Genevant Sciences

Tracy Meffen | VP, Quality & Regulatory Affairs, | Genevant Sciences » speaking at Orphan USA

Pooja Merchant, Head Of Patient Partnership, Engagement And Oncology, Bayer US LLC

Pooja Merchant | Head Of Patient Partnership, Engagement And Oncology | Bayer US LLC » speaking at Orphan USA

Parag Meswani, SVP, Commercial, Axovant Gene Therapies

Parag Meswani | SVP, Commercial | Axovant Gene Therapies » speaking at Orphan USA

Annastasiah Mudiwa Mhaka, Co-Founder, Convenor, President, Alliance for Artificial Intelligence in Healthcare

Annastasiah Mudiwa Mhaka | Co-Founder, Convenor, President | Alliance for Artificial Intelligence in Healthcare » 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

Randy Milby, Chief Executive Officer, Hillstream Biopharma

Randy Milby | Chief Executive Officer | Hillstream Biopharma » 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

Ali Mohamadi, Executive Director, BioMarin Pharmaceutical Inc.

Ali Mohamadi | Executive Director | BioMarin Pharmaceutical Inc. » speaking at Orphan USA

Samarendra Mohanty, Founder And President, NanoScope Technologies

Samarendra Mohanty | Founder And President | NanoScope Technologies » speaking at Orphan USA

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

Michael Murphy | Chief Medical And Scientific Officer | Worldwide Clinical Trials » speaking at Orphan USA

Michelle Muscat, Co-Founder And President, National Alliance for Rare Diseases Support - Malta

Michelle Muscat | Co-Founder And President | National Alliance for Rare Diseases Support - Malta » 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, VP, Regulatory Affairs & Program Management, Optum

Lauren Neighbours | VP, Regulatory Affairs & Program Management | Optum » speaking at Orphan USA

Brian Newkirk, Market Access Director, Alnylam Pharmaceuticals

Brian Newkirk | Market Access Director | Alnylam Pharmaceuticals » speaking at Orphan USA

Jay Newman, Senior Vice President and General Manager, Head Of Us Commercial, Spark Therapeutics

Jay Newman | Senior Vice President and General Manager, Head Of Us Commercial | Spark Therapeutics » speaking at Orphan USA

Scott Nickerson, Senior Vice President, Global Quality, Moderna Therapeutics

Scott Nickerson | Senior Vice President, Global Quality | Moderna Therapeutics » speaking at Orphan USA

Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions

Jan Nielsen | Division President, Patient Solutions | AssistRx Patient Solutions » 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, Senior Vice President Regulatory Affairs, Alnylam Pharmaceuticals

Sara Nochur | Senior Vice President Regulatory Affairs | Alnylam Pharmaceuticals » speaking at Orphan USA

Wolfram Nothaft, Chief Medical Officer, Takeda

Wolfram Nothaft | Chief Medical Officer | Takeda » speaking at Orphan USA

Lynn O'Connor Vos, Chief Executive Officer, Muscular Dystrophy Association

Lynn O'Connor Vos | Chief Executive Officer | Muscular Dystrophy Association » 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, Assessor, AIFA

Arrigo Paciello | Assessor | AIFA » speaking at Orphan USA

Cindy Padgett, Vice President, PharmaCord

Nick Page, Chief Operations Officer, Oxford Biomedica

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

Palani Palaniappan, Head Of Technical Operations And Andover Site, Sarepta Therapeutics

Palani Palaniappan | Head Of Technical Operations And Andover Site | 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

Andrew Parece, Vice President, Charles River Associates

Andrew Parece | Vice President | Charles River Associates » 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 And Public Affairs, Takeda

Linn Parrish | Head of CSR Innovation And Stakeholder Engagement, Corporate Communications And 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

Dmitry Petrov, Head Of Medical Writing And Pharmacovigilance, ICRC-Weyer GmbH

Dmitry Petrov | Head Of Medical Writing And Pharmacovigilance | ICRC-Weyer GmbH » speaking at Orphan USA

Areejit Phukan, Director, Analytics And Forecasting, Insmed Inc

Areejit Phukan | Director, Analytics And Forecasting | Insmed Inc » 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

Sarah Pitluck, Head, Global Pricing And Reimbursement, Spark Therapeutics

Sarah Pitluck | Head, Global Pricing And Reimbursement | Spark Therapeutics » 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

Leslie Powell, Director Of Policy And Advocacy, Cystic Fibrosis Foundation

Leslie Powell | Director Of Policy And Advocacy | Cystic Fibrosis Foundation » speaking at Orphan USA

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

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

Anthony Quinn, President And Chief Executive Officer, Aeglea Biotherapeutics

Anthony Quinn | President And Chief Executive Officer | Aeglea Biotherapeutics » speaking at Orphan USA

Harsha Rajasimha, Founder And Chairman, IndoUSrare

Harsha Rajasimha | Founder And Chairman | IndoUSrare » 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, National Cancer Institute

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

Frederic Revah, Chief Executive Officer, Genethon

Frederic Revah | Chief Executive Officer | Genethon » 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 And U.S. Field Medical Engagement, Ultragenyx Pharmaceutical

Marisa Ricci | Vice President Patient Diagnosis Programs And U.S. Field Medical Engagement | Ultragenyx Pharmaceutical » speaking at Orphan USA

Dara Richardson-Heron, Chief Patient Officer, Pfizer

Dara Richardson-Heron | Chief Patient Officer | Pfizer » 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

Rich Rieger, Managing Director, Life Sciences, Huron Consulting Group

Rich Rieger | Managing Director, Life Sciences | Huron Consulting Group » 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

Kieran Rooney, Vice President, Strategic Alliances And Licensing, Amryt Pharmaceuticals

Kieran Rooney | Vice President, Strategic Alliances And Licensing | Amryt Pharmaceuticals » 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

Ruth Rostron, Vice President Of Project Management, Inceptua Medicines Access

Ruth Rostron | Vice President Of Project Management | Inceptua Medicines Access » 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

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

Kacper Rucinski, Executive Committee Member, SMA Europe

Kacper Rucinski | Executive Committee Member | SMA Europe » speaking at Orphan USA

Christopher Rudolf, Founder And Chief Executive Officer, volv global

Christopher Rudolf | Founder And Chief Executive Officer | volv global » speaking at Orphan USA

David Russell, Head Of External Innovation, Actelion-Janssen

David Russell | Head Of External Innovation | Actelion-Janssen » speaking at Orphan USA

Leo Russo, Senior Director, Epidemiology Worldwide Safety And Regulatory, Pfizer

Leo Russo | Senior Director, Epidemiology Worldwide Safety And Regulatory | Pfizer » 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

Denise Scots Knight, Chief Executive Officer And Co-Founder, Mereo BioPharma

Denise Scots Knight | Chief Executive Officer And Co-Founder | Mereo BioPharma » 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 And Medical Ethics Working Group, Assistant Professor Of Psychiatry, Icahn School Of Medicine, The Mount Sinai Hospital

Lesha Shah | Co-Chair, Pediatric Gene Therapy And 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

Adam Shaywitz, Chief Medical Officer, Rare Disease Drug Development, BridgeBio Pharma

Adam Shaywitz | Chief Medical Officer, Rare Disease Drug Development | BridgeBio 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

Oodaye Shukla, Chief Data Officer, HVH Precision Analytics

Oodaye Shukla | Chief Data Officer | HVH Precision Analytics » speaking at Orphan USA

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

Cornell Stamoran, Vice President of Corporate Strategy, Catalent Pharma Solutions

Cornell Stamoran | Vice President of Corporate Strategy | Catalent Pharma Solutions » speaking at Orphan USA

Kim Stephens, President, Project Alive

Kim Stephens | President | Project Alive » speaking at Orphan USA

Dean Suhr, President, MLD Foundation

Dean Suhr | President | MLD Foundation » 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

Magali Taiel, CMO, GenSight Biologics

Magali Taiel | CMO | GenSight Biologics » speaking at Orphan USA

Jason Tardio, Chief Commercial Officer, Ovid Therapeutics

Jason Tardio | Chief Commercial Officer | Ovid Therapeutics » speaking at Orphan USA

Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences

Ulrich Thienel | Chief Executive Officer | ReAlta Life Sciences » speaking at Orphan USA

Jan Thirkettle, Chief Development Officer, Freeline Therapeutics

Jan Thirkettle | Chief Development Officer | Freeline 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

Cynthia Tifft, Deputy Clinical Director, Office Of The Clinical Director, National Human Genome Research, National Human Genome Research Institute

Cynthia Tifft | Deputy Clinical Director, Office Of The Clinical Director, National Human Genome Research | National Human Genome Research Institute » 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

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

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

Sheela Upadhyaya, Associate Director Hst, National Institute for Health and Care Excellence

Sheela Upadhyaya | Associate Director Hst | National Institute for Health and Care Excellence » speaking at Orphan USA

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

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

Thomas VanCott, Chief Technology Strategy Officer, Catalent Biologics | Paragon Gene Therapy

Thomas VanCott | Chief Technology Strategy Officer | Catalent Biologics | Paragon Gene Therapy » 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

Christina Waters, Chief Executive Officer And Founder, RARE Science

Christina Waters | Chief Executive Officer And Founder | RARE Science » speaking at Orphan USA

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

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

Samuel Agyei Wiafe, Executive Director, Rare Disease Ghana Initiative

Samuel Agyei Wiafe | Executive Director | Rare Disease Ghana Initiative » 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

Jonathan Woodring, Executive Vice President And General Manager, IPM.ai

Jonathan Woodring | Executive Vice President And General Manager | IPM.ai » 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

Zheng Yang, Head Of Technology And Data Innovation, Customer Value, Boehringer Ingelheim

Zheng Yang | Head Of Technology And Data Innovation, Customer Value | Boehringer Ingelheim » 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

Wyatt Yue, Principal Investigator, Associate Professor, Lead Scientist, Rare Disease Research, Oxford University Structural Genomics Consortium

Wyatt Yue | Principal Investigator, Associate Professor, Lead Scientist, Rare Disease Research | Oxford University Structural Genomics Consortium » speaking at Orphan USA

Martin Zagari, Vice President Global Health Economics, Amgen Inc

Martin Zagari | Vice President Global Health Economics | Amgen Inc » speaking at Orphan USA

Shuyang Zhang, Department Of Cardiology, Peking Union Medical College

Shuyang Zhang | Department Of Cardiology | Peking Union Medical College » speaking at Orphan USA

Ran Zheng, Chief Technology Officer, Orchard Therapeutics

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