2019 speakers

 

Jordan Abhold, Project Manager, Medical Analyst, Engage Health

Jordan Abhold | Project Manager, Medical Analyst | Engage Health » 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

Dennis Akkaya, Corporate Development, myTomorrows

Dennis Akkaya | Corporate Development | myTomorrows » speaking at Orphan USA

Amin Hussain Al Amiri, Assistant Undersecretary Of Public Health Policy And Licensing, United Arab Emirates Ministry of Health and Prevention

Amin Hussain Al Amiri | Assistant Undersecretary Of Public Health Policy And Licensing | United Arab Emirates Ministry of Health and Prevention » speaking at Orphan USA

Charlie Albright, Chief Scientific Officer, Editas Medicine

Charlie Albright | Chief Scientific Officer | Editas Medicine » speaking at Orphan USA

Ron Alfa, Senior Vice President, Translational Discovery, Recursion Pharmaceuticals

Ron Alfa | Senior Vice President, Translational Discovery | Recursion Pharmaceuticals » speaking at Orphan USA

Paul Aliu, Global Head of Medical Governance, Chief Medical Office, Novartis

Paul Aliu | Global Head of Medical Governance, Chief Medical Office | Novartis » speaking at Orphan USA

Jeff Allen, President And Chief Executive Officer, Friends of Cancer Research

Jeff Allen | President And Chief Executive Officer | Friends of Cancer Research » speaking at Orphan USA

Oved Amitay, Chief Business Officer, Centogene

Oved Amitay | Chief Business Officer | Centogene » speaking at Orphan USA

Leonela Amoasii, Director, Exonics

Leonela Amoasii | Director | Exonics » speaking at Orphan USA

Dominique Amory, Partner, Nextep, MEDVANCE France

Dominique Amory | Partner | Nextep, MEDVANCE France » speaking at Orphan USA

Alba Ancochea Díaz, Directora Gerente, Spanish Federation of Rare Disease

Alba Ancochea Díaz | Directora Gerente | Spanish Federation of Rare Disease » speaking at Orphan USA

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

Karen Anderson | Senior Director - Medical Affairs And Rare Genetic Diseases | Agios Pharmaceuticals » speaking at Orphan USA

Daniel Anderson, Head Of Commercial Partnerships, Invitae

Daniel Anderson | Head Of Commercial Partnerships | Invitae » speaking at Orphan USA

Patrick Anquetil, Chief Executive Officer and Co Founder, Portal Instruments

Patrick Anquetil | Chief Executive Officer and Co Founder | Portal Instruments » 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

Jamie Arnott, Senior Project Director, Rho, Inc

Jamie Arnott | Senior Project Director | Rho, Inc » 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

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

Imran Babar | Chief Business Officer | Cydan II, Inc. » speaking at Orphan USA

Melinda Bachini, Advocacy Coordinator, The Cholangiocarcinoma Foundation

Melinda Bachini | Advocacy Coordinator | The Cholangiocarcinoma Foundation » 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

Rahul Ballal, Cheif Executive Officer, Imara

Rahul Ballal | Cheif Executive Officer | Imara » speaking at Orphan USA

Mariah Baltezegar, Executive Director Clinical Development, Syneos Health

Mariah Baltezegar | Executive Director Clinical Development | Syneos Health » speaking at Orphan USA

Michael Barry, Clinical Director, National Centre for Pharmacoeconomics

Michael Barry | Clinical Director | National Centre for Pharmacoeconomics » 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

Heather Behanna, Principal, Sofinnova Ventures

Heather Behanna | Principal | Sofinnova Ventures » speaking at Orphan USA

Stuart Bell, Vice President, Consulting, Inceptua Medicines Access

Stuart Bell | Vice President, Consulting | Inceptua Medicines Access » speaking at Orphan USA

Denis Belyakov, Executive Director, Union of Patients' And Patients' Organizations With Rare Diseases

Denis Belyakov | Executive Director | Union of Patients' And Patients' Organizations With Rare Diseases » speaking at Orphan USA

Michelle Berg, Vice President Patient Affairs, Abeona Therapeutics Inc.

Michelle Berg | Vice President Patient Affairs | Abeona Therapeutics Inc. » speaking at Orphan USA

Nirjal Bhattarai, Principal Investigator, Division Of Cellular And Gene Therapies, F.D.A. U.S. Food and Drug Administration

Nirjal Bhattarai | Principal Investigator, Division Of Cellular And Gene Therapies | F.D.A. U.S. Food and Drug Administration » speaking at Orphan USA

Luc Boileau, Président-Directeur General, Inesss

Luc Boileau | Président-Directeur General | Inesss » speaking at Orphan USA

Amanda Bok, Chief Executive Officer, European Haemophillia Cosortium

Amanda Bok | Chief Executive Officer | European Haemophillia Cosortium » speaking at Orphan USA

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

Tim Boyd | Director Of State Policy | National Organization for Rare Disorders » 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

Aoife Brennan, Chief Executive Officer, Synlogic Inc

Aoife Brennan | Chief Executive Officer | Synlogic Inc » speaking at Orphan USA

Brian Bronk, Head Of Business Development, Rare Diseases And Disorders, Sanofi

Brian Bronk | Head Of Business Development, Rare Diseases And Disorders | Sanofi » speaking at Orphan USA

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

Max Bronstein | Senior Director, Health Policy And Corporate Affairs | Audentes Therapeutics » speaking at Orphan USA

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

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

Max Brosa, Owner, Oblikue Consulting, MEDVANCE Spain

Max Brosa | Owner | Oblikue Consulting, MEDVANCE Spain » speaking at Orphan USA

Ailsa Brown, Lead Health Economist, Scottish Medicines Consortium

Ailsa Brown | Lead Health Economist | Scottish Medicines Consortium » 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

Greg Candelmo, Director Of Sales, Clincierge

Greg Candelmo | Director Of Sales | Clincierge » 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

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

Rick Chapman | Director Of Health Economics | Institute for Clinical and Economic Review » speaking at Orphan USA

Maurizio Chiriva, Cheif Executive Officer, Kiromic Llc

Maurizio Chiriva | Cheif Executive Officer | Kiromic Llc » speaking at Orphan USA

Andre Choulika, Chairman And Chief Executive Officer, Cellectis

Andre Choulika | Chairman And Chief Executive Officer | Cellectis » speaking at Orphan USA

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

Christopher Chute | Bloomberg Distinguished Professor Of Health Informatics | Johns Hopkins University » speaking at Orphan USA

Gabriel Cohn, Vice President Of Clinical Development Lead, Avrobio

Alexander Cole, Global Head, Epidemiology, Alexion Pharmaceuticals

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

Jerry Collins, Associate Director, Developmental Therapeutics Program Division Of Cancer Treatment And Diagnosis, National Cancer Institute

Jerry Collins | Associate Director, Developmental Therapeutics Program Division Of Cancer Treatment And Diagnosis | National Cancer Institute » speaking at Orphan USA

Morgan Conn, Chief Business Officer, Pharvaris BV

Morgan Conn | Chief Business Officer | Pharvaris BV » speaking at Orphan USA

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

Linda Cooper | Director Of Special Initiatives | Association For Creatine Deficiencies » speaking at Orphan USA

Michael Cox, Senior Director Of Clinical Development And Medical Affairs, Loxo Oncology Inc

Michael Cox | Senior Director Of Clinical Development And Medical Affairs | Loxo Oncology Inc » speaking at Orphan USA

Stewart Craig, Chief Manufacturing Officer, Orchard Therapeutics

Stewart Craig | Chief Manufacturing Officer | Orchard Therapeutics » speaking at Orphan USA

Jeremy Cress, Director Of Operations, Fibrocell Therapeutics

Brad Crittenden, Executive Director, Canadian Association of Pompe

Brad Crittenden | Executive Director | Canadian Association of Pompe » speaking at Orphan USA

Anne Cropp, Chief Scientific Officer, Early Access Care

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

Gregory Daniel, Deputy Director, Duke Margolis Center for Health Policy

Gregory Daniel | Deputy Director | Duke Margolis Center for Health Policy » speaking at Orphan USA

Cynthia Denemark, Pharmacy Director, Delaware Division of Medicaid & Medical Assistance

Cynthia Denemark | Pharmacy Director | Delaware Division of Medicaid & Medical Assistance » speaking at Orphan USA

Martin Devenport, Chief Operating Officer, OncoImmune Inc

Martin Devenport | Chief Operating Officer | OncoImmune Inc » speaking at Orphan USA

Lisa Dilworth, Vice President Rare And Orphan Diseases, Synteract

Lisa Dilworth | Vice President Rare And Orphan Diseases | Synteract » speaking at Orphan USA

Dan Donovan, Co-Founder And Chief Executive Officer, rareLife solutions

Dan Donovan | Co-Founder And Chief Executive Officer | rareLife solutions » speaking at Orphan USA

Scott Dorfman, Chief Executive Officer, Odylia Therapeutics

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

Johan Drott, Chief Executive Officer, Respiratorius

Johan Drott | Chief Executive Officer | Respiratorius » speaking at Orphan USA

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

Kelly Du Plessis | Chief Executive Officer | Rare Diseases South Africa » speaking at Orphan USA

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

Ashish Dugar | Vice President, Global Medical Affairs | Sarepta Therapeutics » speaking at Orphan USA

Tim Dyer, Chief Executive Officer, Addex Therapeutics

Tim Dyer | Chief Executive Officer | Addex Therapeutics » speaking at Orphan USA

Edith Eby, Vice President, Pfizer

Edith Eby | Vice President | Pfizer » speaking at Orphan USA

Kara Eichelkraut, Senior Manager, Patient Advocacy, Reata Pharmaceuticals

Kara Eichelkraut | Senior Manager, Patient Advocacy | Reata Pharmaceuticals » speaking at Orphan USA

Jaysson Eicholtz, Director Of Gmp Operations, Nationwide Children's Hospital

Jaysson Eicholtz | Director Of Gmp Operations | Nationwide Children's Hospital » speaking at Orphan USA

Khair ElZarrad, Deputy Director, Office of Medical Policy, Center for Drug Evaluation and Research, F.D.A. U.S. Food and Drug Administration

Khair ElZarrad | Deputy Director, Office of Medical Policy, Center for Drug Evaluation and Research | F.D.A. U.S. Food and Drug Administration » speaking at Orphan USA

Patti Engel, Founder, President And Chief Executive Officer, Engage Health

Patti Engel | Founder, President And Chief Executive Officer | Engage Health » speaking at Orphan USA

Wendy Erler, Vice President Of Patient Advocacy And Market Insights, Wave Life Sciences

Wendy Erler | Vice President Of Patient Advocacy And Market Insights | Wave Life Sciences » speaking at Orphan USA

John Evans, Chief Executive Officer, Beam Therapeutics

John Evans | Chief Executive Officer | Beam Therapeutics » speaking at Orphan USA

Vincent Forster, Chief Executive Officer, Versantis

Vincent Forster | Chief Executive Officer | Versantis » speaking at Orphan USA

Jorge Franceschi, Director Business Development, Rx Solutions Inc

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

Roser Francisco Bordas | Head Technician, Rare Disease Program, Catalan Health Service. Spain | CatSalut » speaking at Orphan USA

Rich Franco, Vice President of Clinical Strategy, Eiger Biopharmaceuticals

Rich Franco | Vice President of Clinical Strategy | Eiger Biopharmaceuticals » speaking at Orphan USA

John Freshley, President And Chief Executive Officer, ONL Therapeutics

John Freshley | President And Chief Executive Officer | ONL Therapeutics » speaking at Orphan USA

Sarah Fry, Global Business Development Manager, Inceptua Medicines Access

Ilan Ganot, President and Chief Executive Officer, Solid Biosciences

Ilan Ganot | President and Chief Executive Officer | Solid Biosciences » speaking at Orphan USA

Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders

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

Deborah Gill, Professor Of Gene Medicine, University Of Oxford, Cystic Fibrosis Gene Therapy Consortium

Deborah Gill | Professor Of Gene Medicine, University Of Oxford | Cystic Fibrosis Gene Therapy Consortium » speaking at Orphan USA

Manijeh Goldberg, Chief Executive Officer, Privo Technologies

Manijeh Goldberg | Chief Executive Officer | Privo Technologies » speaking at Orphan USA

Kate Grady, Senior Director, Huron Life Sciences

Kate Grady | Senior Director | Huron Life Sciences » speaking at Orphan USA

Scott Gray, Chief Executive Officer, Clincierge

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

John Gray, Senior Vice President And Chief Scientific Officer, Audentes Therapeutics

John Gray | Senior Vice President And Chief Scientific Officer | Audentes Therapeutics » speaking at Orphan USA

James Greenwood, Chief Executive Officer, Biotechnology Industry Organization

James Greenwood | Chief Executive Officer | Biotechnology Industry Organization » 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

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

Dorit Harati | Vice President Of Quality Assurance | Gamida Cell Ltd » speaking at Orphan USA

Eric Hartman, Executive Director, Choroideremia Research Foundation Inc

Eric Hartman | Executive Director | Choroideremia Research Foundation Inc » speaking at Orphan USA

Christina Hartman, Head of Advocacy & Policy, Everylife Foundation

Christina Hartman | Head of Advocacy & Policy | Everylife Foundation » speaking at Orphan USA

Rachel Haurwitz, President And Chief Executive Officer, Caribou Biosciences

Rachel Haurwitz | President And Chief Executive Officer | Caribou Biosciences » speaking at Orphan USA

Lee Henderson, Chief Executive Officer And Founder, Vybion

Samit Hirawat, Executive Vice President, Head Of Oncology Global Development, Novartis

Samit Hirawat | Executive Vice President, Head Of Oncology Global Development | Novartis » speaking at Orphan USA

Melissa Hogan, Founder And President, Project Alive

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

John Hopper, President And Executive Director, Fibrolamellar Cancer Foundation

John Hopper | President And Executive Director | Fibrolamellar Cancer Foundation » speaking at Orphan USA

Tony Howell, Co-Founder And Chief Operating Officer, rareLife solutions

Tony Howell | Co-Founder And Chief Operating Officer | rareLife solutions » speaking at Orphan USA

Kevin Huang, President, Chinese Organization for Rare Disorders

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

Wills Hughes-Wilson, Head Of Patient Access And Commercial Planning, Mereo BioPharma

Wills Hughes-Wilson | Head Of Patient Access  And Commercial Planning | Mereo BioPharma » speaking at Orphan USA

Timothy Hunt, Senior Vice President, Corporate Affairs, Editas Medicine

Timothy Hunt | Senior Vice President, Corporate Affairs | Editas Medicine » speaking at Orphan USA

Adam Hutchings, Managing Director, Dolon

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

Usman Iqbal, Vice President, Medical Affairs, Acer Therapeutics

Usman Iqbal | Vice President, Medical Affairs | Acer Therapeutics » speaking at Orphan USA

Ilan Irony, Deputy Director Division Of Clinical Evaluation And Pharmacology, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration

Ilan Irony | Deputy Director Division Of Clinical Evaluation And Pharmacology, Center for Biologics Evaluation and Research (CBER) | Food and Drug Administration » speaking at Orphan USA

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

Eugean Jiwanmall | Senior Research Analyst, Technology Evaluation And Medical Policy | Independence Blue Cross » speaking at Orphan USA

Steve Kagan, Senior Director, Rare Disease Medical Affairs, Pfizer

Steve Kagan | Senior Director, Rare Disease Medical Affairs | Pfizer » speaking at Orphan USA

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

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

Matthew Kalnik, Chief Executive Officer, Antidote Therapeutics

Vikram Karnani, Chief Commercial Officer, Horizon Pharma

Vikram Karnani | Chief Commercial Officer | Horizon Pharma » speaking at Orphan USA

Vipul Kashyap, Chief Information Architect, Northwell Health

Vipul Kashyap | Chief Information Architect | Northwell Health » speaking at Orphan USA

Manohar Katakam, Founder And Chief Executive Officer, Sterotherapeutics

Manohar Katakam | Founder And Chief Executive Officer | Sterotherapeutics » speaking at Orphan USA

Barrett Katz, Chief Medical Officer, GenSight Biologics

Barrett Katz | Chief Medical Officer | GenSight Biologics » speaking at Orphan USA

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

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

Annie Kennedy, Senior Vice President Of Legislation And Public Policy, Parent Project Muscular Dystrophy

Annie Kennedy | Senior Vice President Of Legislation And Public Policy | Parent Project Muscular Dystrophy » speaking at Orphan USA

Behrang Kianzad, Faculty, Centre For Advanced Studies In Biomedical Innovation Law, University of Copenhagen

Behrang Kianzad | Faculty, Centre For Advanced Studies In Biomedical Innovation Law | University of Copenhagen » speaking at Orphan USA

Terri Klein, President And Chief Executive Officer, National M.P.S. Society

Terri Klein | President And Chief Executive Officer | National M.P.S. Society » speaking at Orphan USA

Daniel Knecht, Vice President, Clinical Strategy And Policy, Aetna

Daniel Knecht | Vice President, Clinical Strategy And Policy | Aetna » speaking at Orphan USA

Bridget Knisely, MFA, Project Manager, Engage Health

Bridget Knisely | MFA, Project Manager | Engage Health » speaking at Orphan USA

Simon Kos, Chief Medical Officer, Microsoft

Simon Kos | Chief Medical Officer | Microsoft » speaking at Orphan USA

Walter Kowtoniuk, Principle, Third Rock Ventures, LLC

Walter Kowtoniuk | Principle | Third Rock Ventures, LLC » speaking at Orphan USA

Michelle Krishnan, Translational Medicine Leader In Rare Diseases, Roche

Michelle Krishnan | Translational Medicine Leader In Rare Diseases | Roche » speaking at Orphan USA

Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

Samarth Kulkarni | Chief Executive Officer | CRISPR Therapeutics » speaking at Orphan USA

Steven Laffoon, President, Wylder Nation

Steven Laffoon | President | Wylder Nation » 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

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

Ray Lee, Founder And Chief Medical Officer, Teclison Limited

Ray Lee | Founder And Chief Medical Officer | Teclison Limited » speaking at Orphan USA

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

Thomas Lester | Senior Director Of Product Development | BioMarin Pharmaceutical Inc. » speaking at Orphan USA

Paul Levesque, Global President, Rare Disease, Pfizer

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

André Liamas, Director, Market Access and Government Affairs, Biogen

André Liamas | Director, Market Access and Government Affairs | Biogen » speaking at Orphan USA

Edmund Lim, Co-Founder, We CARE Journey

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

Sook Yee Lim, Co-Founder And President, We CARE Journey

Sook Yee Lim | Co-Founder And President | We CARE Journey » speaking at Orphan USA

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

Julie Lin | Global Project Head, Early Clinical And Business Development | Sanofi Genzyme Corp » speaking at Orphan USA

Qing Liu, Head of Biostatistics and Data Management, Amicus Therapeutics Inc

Qing Liu | Head of Biostatistics and Data Management | Amicus Therapeutics Inc » speaking at Orphan USA

Allyson Lock, President, NEW ZEALAND POMPE NETWORK

Allyson Lock | President | NEW ZEALAND POMPE NETWORK » speaking at Orphan USA

Robert Long, Executive Director Of Strategic Development, Uplifting Athletes

Isabelle Lousada, President And Chief Executive Officer, Amyloidosis Research Consortium

Isabelle Lousada | President And Chief Executive Officer | Amyloidosis Research Consortium » speaking at Orphan USA

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

Patrick Lu | President And Chief Executive Officer | Sirnaomics, Inc. » speaking at Orphan USA

Sanjeev Luther, Chief Executive Officer, Rafael Pharmaceuticals

Sanjeev Luther | Chief Executive Officer | Rafael Pharmaceuticals » 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

John Maraganore, Chief Executive Officer, Alnylam Pharmaceuticals

John Maraganore | Chief Executive Officer | Alnylam Pharmaceuticals » 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

Kevin Marsh, Executive Director, Patient-Centered Research, Evidera – Evidence, Value & Access by PPD

Kevin Marsh | Executive Director, Patient-Centered Research | Evidera – Evidence, Value & Access by PPD » speaking at Orphan USA

Sebastien Martel, Senior Vice President and Global Head of Rare Diseases, Sanofi Genzyme Corp

Sebastien Martel | Senior Vice President and Global Head of Rare Diseases | Sanofi Genzyme Corp » speaking at Orphan USA

Tara Mathiesen, Director, Us Diagnostics, Orchard Therapeutics

Tara Mathiesen | Director, Us Diagnostics | Orchard Therapeutics » speaking at Orphan USA

Molly Mccarthy, National Director, Us Provider Industry And Chief Nursing Officer, Microsoft

Molly Mccarthy | National Director, Us Provider Industry And Chief Nursing Officer | Microsoft » speaking at Orphan USA

Susan McCune, Director, Office Of Pediatric Therapeutics, Office Of The Commissioner, U.S. Food and Drug Administration

Susan McCune | Director, Office Of Pediatric Therapeutics, Office Of The Commissioner | U.S. Food and Drug Administration » speaking at Orphan USA

Jenn Mcnary, Duchenne Parent Advocate, Consultant

Jenn Mcnary | Duchenne Parent Advocate | Consultant » speaking at Orphan USA

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

Paul Melmeyer | Associate Director Of Public Policy | National Organization for Rare Disorders » speaking at Orphan USA

Debra Miller, Founder And President, CureDuchenne

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

Chris Moen, President, Choroideremia Research Foundation Inc

Chris Moen | President | Choroideremia Research Foundation Inc » speaking at Orphan USA

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

Juliet Moritz | Executive Director, Program Strategy, Rare Disease And Pediatrics/Patient Engagement | Premier Research » speaking at Orphan USA

Trista Morrison, Vice President Of Communications And Patient Advocacy, North America, Sobi

Trista Morrison | Vice President Of Communications And Patient Advocacy, North America | Sobi » 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

Anant Murthy, Vice President Of Market Access And Pricing, Alnylam

Anant Murthy | Vice President Of Market Access And Pricing | Alnylam » speaking at Orphan USA

Joseph Musumeci, President, BluePrint Orphan

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

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

Francois Nader | Board Chairman, Acceleron Pharma (Xlrn), Former President And Chief Executive Officer | NPS Pharmaceuticals Inc » speaking at Orphan USA

Alexander Natz, Secretary General, EUCOPE

Alexander Natz | Secretary General | EUCOPE » speaking at Orphan USA

Knut Niss, Chief Technology Officer, MustangBio

Knut Niss | Chief Technology Officer | MustangBio » speaking at Orphan USA

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

Megan O'Boyle | Principal Investigator | Phelan-McDermid Syndrome Foundation » speaking at Orphan USA

Tuyen Ong, Chief Development Officer, Nightstar Therapeutics

Tuyen Ong | Chief Development Officer | Nightstar Therapeutics » speaking at Orphan USA

Corrie Painter, Associate Director, Count Me In

Corrie Painter | Associate Director | Count Me In » speaking at Orphan USA

Jim Palma, Executive Director, TargetCancer Foundation

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

Clark Paramore, Head Of Value Demonstration, bluebird bio

Clark Paramore | Head Of Value Demonstration | bluebird bio » 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 Corporate Responsibility, Shire

Linn Parrish | Head Of Corporate Responsibility | Shire » speaking at Orphan USA

Gina Parziale, Executive Director, Alport Syndrome Foundation

Gina Parziale | Executive Director | Alport Syndrome Foundation » speaking at Orphan USA

Nita Patel, Senior Director, Patient & Professional Advocacy, Amicus Therapeutics Inc

Nita Patel | Senior Director, Patient & Professional Advocacy | Amicus Therapeutics Inc » speaking at Orphan USA

Matthew Patterson, President And Chief Executive Officer, Audentes Therapeutics

Matthew Patterson | President And Chief Executive Officer | Audentes Therapeutics » speaking at Orphan USA

Malinda M Peeples, Vice President of Clinical Services Programs and Research, WellDoc

Malinda M Peeples | Vice President of Clinical Services Programs and Research | WellDoc » speaking at Orphan USA

Stuart Peltz, Chief Executive Officer, PTC Therapeutics

Stuart Peltz | Chief Executive Officer | PTC Therapeutics » speaking at Orphan USA

Steven Petrou, Chief Scientific Officer, RogCon

Steven Petrou | Chief Scientific Officer | RogCon » speaking at Orphan USA

Doug Pfaff, Managing Director, Huron Life Sciences

Doug Pfaff | Managing Director | Huron Life Sciences » speaking at Orphan USA

Han Phan, Founder And Executive Officer, Rare Disease Research

Han Phan | Founder And Executive Officer | Rare Disease Research » speaking at Orphan USA

Mathew Pletcher, Head Of Rare Disease Discovery, Roche

Mathew Pletcher | Head Of Rare Disease Discovery | Roche » speaking at Orphan USA

Mike Porath, Founder And Chief Executive Officer, The Mighty

Mike Porath | Founder And Chief Executive Officer | The Mighty » speaking at Orphan USA

Antonio Porras, Scientist, Children's National Health System

Antonio Porras | Scientist | Children's National Health System » speaking at Orphan USA

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

Tanya Potashnik | Director Policy And Economic Analysis, Patented Medicine Prices Review Board | Goverment Of Canada » speaking at Orphan USA

Mariangela Prada, Head Of Patient Access Unit, Intexo, MEDVANCE Italy

Mariangela Prada | Head Of Patient Access Unit | Intexo, MEDVANCE Italy » speaking at Orphan USA

Suyash Prasad, Senior Vice President And Chief Medical Officer, Audentes Therapeutics

Suyash Prasad | Senior Vice President And Chief Medical Officer | Audentes Therapeutics » speaking at Orphan USA

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

Sarah Procario | Advocacy-Communications Manager | The Hemophilia Foundation of Michigan » speaking at Orphan USA

Mingxin Qian, Chief Executive Officer, Tongli Biomedical

Mingxin Qian | Chief Executive Officer | Tongli Biomedical » speaking at Orphan USA

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

Janet Rae | Vice President Of Global Head Of Regulatory Affairs | Ultragenyx Pharmaceutical » 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

Julie Raskin, Executive Director, Congenital Hyperinsulinism International

Julie Raskin | Executive Director | Congenital Hyperinsulinism International » 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

Kaye Robertson, Assistant Director, Australian Government Department of Health

Kaye Robertson | Assistant Director | Australian Government Department of Health » speaking at Orphan USA

Susan Robinson, Senior Vice President of Business Development, AnovoRx Group Llc

Susan Robinson | Senior Vice President of Business Development | AnovoRx Group Llc » speaking at Orphan USA

Arndt Rolfs, Chief Executive Officer, Centogene

Arndt Rolfs | Chief Executive Officer | Centogene » speaking at Orphan USA

Kieran Rooney, Vice President Of Strategic Alliances And Licensing, Amryt Pharma

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

Jerome Rossert, Vice President Clinical Development, Vertex Pharmaceuticals

Jerome Rossert | Vice President Clinical Development | Vertex Pharmaceuticals » speaking at Orphan USA

Theodore Roth, Managing Director/Phd Student, University of California, San Francisco

Theodore Roth | Managing Director/Phd Student | University of California, San Francisco » 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

Marco Ruella, Professor of Medicine, Scientific Director, Lymphoma Program Division of Hematology and Oncology Center, University of Pennsylvania

Marco Ruella | Professor of Medicine, Scientific Director, Lymphoma Program Division of Hematology and Oncology Center | University of Pennsylvania » speaking at Orphan USA

Herbert Runnels, Research Fellow And Gene Therapy Global Analytical Lead, Pfizer

Leandro Safatle, Secretário-Executivo, ANVISA

Leandro Safatle | Secretário-Executivo | ANVISA » speaking at Orphan USA

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

Peter Saltonstall | President And Chief Executive Officer | National Organization for Rare Disorders » speaking at Orphan USA

Lori Sames, Founder And President, Hannah's Hope Fund for GAN

Lori Sames | Founder And President | Hannah's Hope Fund for GAN » speaking at Orphan USA

Daniela Santiesteban, Business Development Manager, Salarius Pharmaceuticals

Daniela Santiesteban | Business Development Manager | Salarius Pharmaceuticals » speaking at Orphan USA

Mary Schaheen, President and Director, Prevail Partners

Lee Schalop, Chief Operating Officer, Oncoceutics Inc

Lee Schalop | Chief Operating Officer | Oncoceutics Inc » 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

Jaci Schmidt, Database Administrator, Engage Health

Jaci Schmidt | Database Administrator | Engage Health » 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

Samir Shaikh, Deputy Director, Patient Affairs Staff Office Of The Commissioner, Office Of Medical Products And Tobacco, F.D.A. U.S. Food and Drug Administration

Samir Shaikh | Deputy Director, Patient Affairs Staff Office Of The Commissioner, Office Of Medical Products And Tobacco | F.D.A. U.S. Food and Drug Administration » speaking at Orphan USA

Jeffrey Sherman, Chief Medical Officer, Horizon Pharma

Jeffrey Sherman | Chief Medical Officer | Horizon Pharma » speaking at Orphan USA

Alex Sherman, Director, Center For Innovation And Bioinformatics, Massachusetts General Hospital Neurological Clinical Research Institute

Alex Sherman | Director, Center For Innovation And Bioinformatics | Massachusetts General Hospital Neurological Clinical Research Institute » speaking at Orphan USA

Oodaye Shukla, Chief Data & Analytics Officer, HVH Precision Analytics

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

Patty Sibley, Senior Medical Science Liaison, Enzyvant Therapeutics

Patty Sibley | Senior Medical Science Liaison | Enzyvant Therapeutics » 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

Josh Sommer, Co Founder And Executive Director, Chordoma Foundation

Josh Sommer | Co Founder And Executive Director | Chordoma Foundation » speaking at Orphan USA

Frank Sorgi, President And Chief Executive Officer, FLAG Therapeutics

Frank Sorgi | President And Chief Executive Officer | FLAG Therapeutics » speaking at Orphan USA

David Sourdive, Executive Vice President Technical Operations, Cellectis

David Sourdive | Executive Vice President Technical Operations | Cellectis » 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

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

Jennifer Thornton | Executive Director | A.T. Childrens Project » speaking at Orphan USA

Barry Ticho, Chief Medical Officer, Stoke Therapeutics

Barry Ticho | Chief Medical Officer | Stoke Therapeutics » speaking at Orphan USA

Julie Tierney, Senior Policy Advisor For Strategic Planning And Legislation,Center for Biologics Evaluation and Research (CBER), F.D.A. U.S. Food and Drug Administration

Julie Tierney | Senior Policy Advisor For Strategic Planning And Legislation,Center for Biologics Evaluation and Research (CBER) | F.D.A. U.S. Food and Drug Administration » speaking at Orphan USA

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

Dan Tierno | Strategic Implementation Manager, Global Data Sciences And Analytics | Bayer » 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

Laura Trutoiu, Director Of Research, Association For Creatine Deficiencies

Laura Trutoiu | Director Of Research | Association For Creatine Deficiencies » speaking at Orphan USA

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

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

Tiina Urv, Program Director, Office Of Rare Disease Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)

Tiina Urv | Program Director, Office Of Rare Disease Research, National Center For Advancing Translational Sciences - Ncats | National Institutes of Health (NIH) » speaking at Orphan USA

Goedele Van Haasteren, Hta Specialist, Bundesamt fur Gesundheit

Goedele Van Haasteren | Hta Specialist | Bundesamt fur Gesundheit » speaking at Orphan USA

Ray Veeraraghavan, Director Of It And Informatics, Rady Children�s Institute For Genomic Medicine

Ray Veeraraghavan | Director Of It And Informatics | Rady Children�s Institute For Genomic Medicine » speaking at Orphan USA

Margaret Vernon, Vice-President and General Manager, Patient-Centered Research, Evidera – Evidence, Value & Access by PPD

Margaret Vernon | Vice-President and General Manager, Patient-Centered Research | Evidera – Evidence, Value & Access by PPD » speaking at Orphan USA

Alain Vicari, Chief Executive Officer And Founder, Calypso Biotech

Alain Vicari | Chief Executive Officer And Founder | Calypso Biotech » speaking at Orphan USA

Stefan Walzer, General Manager And Founder, MArS Market Access & Pricing Strategy, MEDVANCE Germany

Stefan Walzer | General Manager And Founder | MArS Market Access & Pricing Strategy, MEDVANCE Germany » speaking at Orphan USA

Jay Weaver, Divisional Vice President, Enterprise Pharmacy, Blue Shield of Illinois, Montana, New Mexico, Oklahoma & Texas

Jay Weaver | Divisional Vice President, Enterprise Pharmacy | Blue Shield of Illinois, Montana, New Mexico, Oklahoma & Texas » speaking at Orphan USA

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

Monica Weldon | Chief Executive Officer | Bridge the Gap -SYNGAP Educatrion and Research Foundation » speaking at Orphan USA

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

James Wilson | Director Of Gene Therapy Program | University of Pennsylvania School Of Medicine » speaking at Orphan USA

Durhane Wong-Rieger, President, Canadian Organization For Rare Disorders

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

Laura Wuerth, Executive Vice President, rareLife solutions

Laura Wuerth | Executive Vice President | rareLife solutions » speaking at Orphan USA

Joe Young, Chief Business Officer, Araim Pharmaceuticals Inc

Joe Young | Chief Business Officer | Araim Pharmaceuticals Inc » speaking at Orphan USA

Martine Zimmermann, Global Head Of Regulatory Affairs, Alexion Pharmaceuticals

Martine Zimmermann | Global Head Of Regulatory Affairs | Alexion Pharmaceuticals » speaking at Orphan USA