2022 Speakers

 

Dennis Akkaya, Chief Commercial Officer, myTomorrows

Dennis Akkaya | Chief Commercial Officer | myTomorrows » speaking at Orphan Drug Congress

Faraz Ali, Chief Executive Officer, Tenaya Therapeutics

Faraz Ali | Chief Executive Officer | Tenaya Therapeutics » speaking at Orphan Drug Congress

Paul Aliu, Head Global Governance Office, Novartis Chief Medical Office (CMO), Novartis

Paul Aliu | Head Global Governance Office, Novartis Chief Medical Office (CMO) | Novartis » speaking at Orphan Drug Congress

Simon Andrews, Senior Vice President, Head of Analytics, EVERSANA

Simon Andrews | Senior Vice President, Head of Analytics | EVERSANA » speaking at Orphan Drug Congress

Samer Ansari, Head of Data, Digital & Technology, Takeda Oncology, Takeda

Samer Ansari | Head of Data, Digital & Technology, Takeda Oncology | Takeda » speaking at Orphan Drug Congress

Derek Ansel, Senior Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials

Derek Ansel | Senior Director, Therapeutic Strategy Lead, Rare Diseases | Worldwide Clinical Trials » speaking at Orphan Drug Congress

Catherine Argyriou, Postdoctoral Fellow, McGill University

Catherine Argyriou | Postdoctoral Fellow | McGill University » speaking at Orphan Drug Congress

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

Francis Arickx | Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy | National Institute for Health and Disability Insurance » speaking at Orphan Drug Congress

Lisa Bacon, Senior Director, Enzyvant Therapeutics

Lisa Bacon | Senior Director | Enzyvant Therapeutics » speaking at Orphan Drug Congress

Ella Balasa, Rare Disease, Patient Advocate

Ella Balasa | Rare Disease | Patient Advocate » speaking at Orphan Drug Congress

Peter Bannister, VP, Life Sciences, Ada Health

Peter Bannister | VP, Life Sciences | Ada Health » speaking at Orphan Drug Congress

Julie Batista, Director of Epidemiology and Biostatistics, Sanofi Genzyme

Julie Batista | Director of Epidemiology and Biostatistics | Sanofi Genzyme » speaking at Orphan Drug Congress

Katherine Beaverson, Executive Director and Head of Patient Advocacy and External Engagement, Rare Disease Research Unit,, Pfizer

Katherine Beaverson | Executive Director and Head of Patient Advocacy and External Engagement, Rare Disease Research Unit, | Pfizer » speaking at Orphan Drug Congress

Stuart Bell, Vice President, Consulting, Inceptua

Stuart Bell | Vice President, Consulting | Inceptua » speaking at Orphan Drug Congress

Joseph Bender, Head of Global Rare Disease Registries, Sanofi Genzyme

Joseph Bender | Head of Global Rare Disease Registries | Sanofi Genzyme » speaking at Orphan Drug Congress

Chris Benevides, Associate Director, Strategic Solutions, Elligo Health Research

Chris Benevides | Associate Director, Strategic Solutions | Elligo Health Research » speaking at Orphan Drug Congress

Alon Ben-Noon, Chief Executive Officer, NeuroSense Therapeutics

Alon Ben-Noon | Chief Executive Officer | NeuroSense Therapeutics » speaking at Orphan Drug Congress

Wendy Benson, Chief Strategy and Innovation Officer, Rady Children's Institute for Genomic Medicine

Wendy Benson | Chief Strategy and Innovation Officer | Rady Children's Institute for Genomic Medicine » speaking at Orphan Drug Congress

Allyson Berent, Chief Science Officer, Foundation for Angelman Therapeutics, COO and Co-Founder, GeneTx Biotherapeutics

Allyson Berent | Chief Science Officer, Foundation for Angelman Therapeutics, COO and Co-Founder | GeneTx Biotherapeutics » speaking at Orphan Drug Congress

Scott Bergeron, Partner, Clinical Research, iTakeControl, a Red Nucleus Company

Scott Bergeron | Partner, Clinical Research | iTakeControl, a Red Nucleus Company » speaking at Orphan Drug Congress

Terry Jo Bichell, Founder And Director, COMBINEDBrain

Terry Jo Bichell | Founder And Director | COMBINEDBrain » speaking at Orphan Drug Congress

Gus Bilirakis, Rare Disease Caucus Lead, United States Congress

Gus Bilirakis | Rare Disease Caucus Lead | United States Congress » speaking at Orphan Drug Congress

Nadia Bodkin, OLM DEI Expert, Rare Advocacy Movement

Nadia Bodkin | OLM DEI Expert | Rare Advocacy Movement » speaking at Orphan Drug Congress

Nicole Boice, Founder & Executive Director, RARE-X

Nicole Boice | Founder & Executive Director | RARE-X » speaking at Orphan Drug Congress

Natasha Bonhomme, Chief Strategy Officer, Genetic Alliance Founder, Expecting Health

Natasha Bonhomme | Chief Strategy Officer, Genetic Alliance Founder | Expecting Health » speaking at Orphan Drug Congress

Simone Boselli, Director, Public Affairs, EURORDIS

Simone Boselli | Director, Public Affairs | EURORDIS » speaking at Orphan Drug Congress

Jason Bradt, Executive Director of Clinical Research, Pharming Healthcare

Jason Bradt | Executive Director of Clinical Research | Pharming Healthcare » speaking at Orphan Drug Congress

Larry Bressler, Head of Value and Market Access, Chiesi Global Rare Diseases

Larry Bressler | Head of Value and Market Access | Chiesi Global Rare Diseases » speaking at Orphan Drug Congress

Phillip Brooks, Acting Director, Office of Rare Diseases Research, NIH - NCATS

Phillip Brooks | Acting Director, Office of Rare Diseases Research | NIH - NCATS » speaking at Orphan Drug Congress

Amy Brower, US National Director, Newborn Screening Translational Research Network (NBSTRN),, American College of Medical Genetics and Genomics

Amy Brower | US National Director, Newborn Screening Translational Research Network (NBSTRN), | American College of Medical Genetics and Genomics » speaking at Orphan Drug Congress

Robert M. Califf, Commissioner, U.S. Food and Drug Administration

Robert M. Califf | Commissioner | U.S. Food and Drug Administration » speaking at Orphan Drug Congress

Jason Cameron, Chief Operating Officer, Orphan Drug Consulting

Jason Cameron | Chief Operating Officer | Orphan Drug Consulting » speaking at Orphan Drug Congress

Katherine Capperella, VP of Global Patient Engagement, Office of the Chief Medical Officer,, Janssen Pharmaceutical companies of Johnson & Johnson

Katherine Capperella | VP of Global Patient Engagement, Office of the Chief Medical Officer, | Janssen Pharmaceutical companies of Johnson & Johnson » speaking at Orphan Drug Congress

Elena Cardenal, Scientific Director, Dravet Syndrome Foundation Spain

Elena Cardenal | Scientific Director | Dravet Syndrome Foundation Spain » speaking at Orphan Drug Congress

Nicole Carswell, Executive Director, Patient Engagement and Recruitment,, Premier Research

Nicole Carswell | Executive Director, Patient Engagement and Recruitment, | Premier Research » speaking at Orphan Drug Congress

Grant Castor, Vice President Commercial Operations, Sentynl Therapeutics

Grant Castor | Vice President Commercial Operations | Sentynl Therapeutics » speaking at Orphan Drug Congress

Jessica CerulloMerrill, Vice President, VOZ Advisors

Jessica CerulloMerrill | Vice President | VOZ Advisors » speaking at Orphan Drug Congress

Anne-Sophie Chalandon, Global Rare Disease Policy Head, Sanofi Genzyme

Anne-Sophie Chalandon | Global Rare Disease Policy Head | Sanofi Genzyme » speaking at Orphan Drug Congress

Corin Chapman, Director, GFPD Board of Directors

Corin Chapman | Director | GFPD Board of Directors » speaking at Orphan Drug Congress

Lynsey Chediak, Head of Partnerships, Rare Base

Lynsey Chediak | Head of Partnerships | Rare Base » speaking at Orphan Drug Congress

Melissa Coloton, EVP, Business Development and Marketing, Scout Clinical

Melissa Coloton | EVP, Business Development and Marketing | Scout Clinical » speaking at Orphan Drug Congress

Jason Colquitt, Chief Executive Officer, Across Healthcare

Jason Colquitt | Chief Executive Officer | Across Healthcare » speaking at Orphan Drug Congress

Michele de Guise, Scientific Vice-President, INESSS

Michele de Guise | Scientific Vice-President | INESSS » speaking at Orphan Drug Congress

Tom Defay, Deputy Head Diagnostics Strategy & Development, Alexion Pharmaceuticals

Tom Defay | Deputy Head Diagnostics Strategy & Development | Alexion Pharmaceuticals » speaking at Orphan Drug Congress

Francisco Diaz-Mitoma, CMO, VBI Vaccines

Francisco Diaz-Mitoma | CMO | VBI Vaccines » speaking at Orphan Drug Congress

Chowdary Dondapati, Executive Director, US Head of Diagnostic Marketing, Precision Medicine, BAYER

Chowdary Dondapati | Executive Director, US Head of Diagnostic Marketing, Precision Medicine | BAYER » speaking at Orphan Drug Congress

Danielle Dong, Global Operations and Advocacy Lead, Rare Disease Registries, Sanofi Genzyme

Danielle Dong | Global Operations and Advocacy Lead, Rare Disease Registries | Sanofi Genzyme » speaking at Orphan Drug Congress

Robert Donnell, Executive Vice President, Medicines Access, Smartway Pharma

Robert Donnell | Executive Vice President, Medicines Access | Smartway Pharma » speaking at Orphan Drug Congress

Marcus Droege, Vice President, Global Value & Market Access, Passage Bio

Marcus Droege | Vice President, Global Value & Market  Access | Passage Bio » speaking at Orphan Drug Congress

Stephanie Duffy, Global Patient Advocacy and Engagement Director, The Janssen Pharmaceutical Company of Johnson and Johnson

Stephanie Duffy | Global Patient Advocacy and Engagement Director | The Janssen Pharmaceutical Company of Johnson and Johnson » speaking at Orphan Drug Congress

Sanjoy Dutta, Chief Scientific Officer, JDRF

Sanjoy Dutta | Chief Scientific Officer | JDRF » speaking at Orphan Drug Congress

Sean Ekins, Founder And Chief Executive Officer, Collaborations Pharmaceuticals

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

Matthew Ellinwood, Chief Scientific Officer, National MPS Society

Matthew Ellinwood | Chief Scientific Officer | National MPS Society » speaking at Orphan Drug Congress

Terri Ellsworth, Consultant, myTomorrows

Terri Ellsworth | Consultant | myTomorrows » speaking at Orphan Drug Congress

Izaskun Elorza, Senior Medical Director,, Parexel

Izaskun Elorza | Senior Medical Director, | Parexel » speaking at Orphan Drug Congress

Wendy Erler, Vice President, Global Head Patient Experience & Insights, Alexion AstraZeneca Rare Disease

Wendy Erler | Vice President, Global Head Patient Experience & Insights | Alexion AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Jorge Faccinetti, Chairman and Chief Executive Officer, Pituitary World News

Jorge Faccinetti | Chairman and Chief Executive Officer | Pituitary World News » speaking at Orphan Drug Congress

Lynn Fallon, President, CTI Clinical Trial and Consulting

Lynn Fallon | President | CTI Clinical Trial and Consulting » speaking at Orphan Drug Congress

Qadan Farah, Clinical Pharmacist Researcher, Curant Health

Qadan Farah | Clinical Pharmacist Researcher | Curant Health » speaking at Orphan Drug Congress

Sarah Farr, Associate, Genesys Capital Partners

Sarah Farr | Associate | Genesys Capital Partners » speaking at Orphan Drug Congress

Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc

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

James Fink, Senior Principal Scientist, Q-State Biosciences

James Fink | Senior Principal Scientist | Q-State Biosciences » speaking at Orphan Drug Congress

Aliza Fink, Director of Research Programs, National Organization for Rare Disorders (NORD)

Aliza Fink | Director of Research Programs | National Organization for Rare Disorders (NORD) » speaking at Orphan Drug Congress

Ryan Fischer, Chief Advocacy Officer, Parent Project Muscular Dystrophy

Ryan Fischer | Chief Advocacy Officer | Parent Project Muscular Dystrophy » speaking at Orphan Drug Congress

Daniel Fischer, President and Chief Executive Officer, Tevard Biosciences

Daniel Fischer | President and Chief Executive Officer | Tevard Biosciences » speaking at Orphan Drug Congress

Al Freedman, Counseling Psychologist, Freedman Counseling Associates

Al Freedman | Counseling Psychologist | Freedman Counseling Associates » speaking at Orphan Drug Congress

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 Drug Congress

Milda Galkute, Researcher, Spanish Federation of Rare Disease

Milda Galkute | Researcher | Spanish Federation of Rare Disease » speaking at Orphan Drug Congress

Nicolas Garnier, Senior Director Patient Advocacy Lead, Pfizer

Nicolas Garnier | Senior Director Patient Advocacy Lead | Pfizer » speaking at Orphan Drug Congress

Safiyya Gassman, Senior Director, Policy & Public Affairs, Rare Disease & Gene Therapy, Pfizer

Safiyya Gassman | Senior Director, Policy & Public Affairs, Rare Disease & Gene Therapy | Pfizer » speaking at Orphan Drug Congress

Joseph Geraci, Chief Scientific Officer, Nurosene Health Inc.

Joseph Geraci | Chief Scientific Officer | Nurosene Health Inc. » speaking at Orphan Drug Congress

Lev Gerlovin, Vice President, Life Sciences Practice, Charles River Associates

Lev Gerlovin | Vice President, Life Sciences Practice | Charles River Associates » speaking at Orphan Drug Congress

Diego Fernando Gil Cardozo, Executive Director, Federación Colombiana de Enfermedades Raras -FECOER-

Diego Fernando Gil Cardozo | Executive Director | Federación Colombiana de Enfermedades Raras -FECOER- » speaking at Orphan Drug Congress

Christoph Glaetzer, Chief Global Value and Access Officer, Janssen

Christoph Glaetzer | Chief Global Value and Access Officer | Janssen » speaking at Orphan Drug Congress

Sarah Glass, Chief Development Officer, n-Lorem Foundation

Sarah Glass | Chief Development Officer | n-Lorem Foundation » speaking at Orphan Drug Congress

Richard Gliklich, Chief Executive Officer, OM1

Richard Gliklich | Chief Executive Officer | OM1 » speaking at Orphan Drug Congress

Deb Gouveia, SVP Development Operations, Triplet Therapeutics

Deb Gouveia | SVP Development Operations | Triplet Therapeutics » speaking at Orphan Drug Congress

Scott Gray, Chief Executive Officer, Clincierge

Scott Gray | Chief Executive Officer | Clincierge » speaking at Orphan Drug Congress

Fumie Griego, Deputy Director General And Chief Operating Officer, International Federation of Pharmaceutical Manufacturers and Associations

Fumie Griego | Deputy Director General And Chief Operating Officer | International Federation of Pharmaceutical Manufacturers and Associations » speaking at Orphan Drug Congress

Edith Gross, Health and Social Science Senior Project Manager, EURORDIS

Edith Gross | Health and Social Science Senior Project Manager | EURORDIS » speaking at Orphan Drug Congress

Abhi Gupta, Senior Director, Global Gene Therapy, Pfizer

Abhi Gupta | Senior Director, Global Gene Therapy | Pfizer » speaking at Orphan Drug Congress

Pamela Guthrie, Director, Sales and Marketing, Clincierge

Pamela Guthrie | Director, Sales and Marketing | Clincierge » speaking at Orphan Drug Congress

Elin Haf Davies, Chief Executive Officer, Aparito

Elin Haf Davies | Chief Executive Officer | Aparito » speaking at Orphan Drug Congress

Sam Hall, Managing Partner, Undisclosed Venture Fund

Sam Hall | Managing Partner | Undisclosed Venture Fund » speaking at Orphan Drug Congress

Michael Hamper, Medical/Science Advisory, Kleine-Levin Syndrome Foundation

Michael Hamper | Medical/Science Advisory | Kleine-Levin Syndrome Foundation » speaking at Orphan Drug Congress

Christopher Hamper, Medical Advisory Board Member, Kleine-Levin Syndrome Foundation

Christopher Hamper | Medical Advisory Board Member | Kleine-Levin Syndrome Foundation » speaking at Orphan Drug Congress

Guido Hartmann, Head of Translational Pharmacology, Centogene Switzerland AG

Guido Hartmann | Head of Translational Pharmacology | Centogene Switzerland AG » speaking at Orphan Drug Congress

Scott Harwood, Director, Check Rare

Scott Harwood | Director | Check Rare » speaking at Orphan Drug Congress

Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics

Shane Hegarty | Chief Scientific Officer & Co-Founder | AXONIS Therapeutics » speaking at Orphan Drug Congress

Rachele Hendricks-Sturrup, Research Director, Real World Evidence, Duke Margolis Center for Health Policy

Rachele Hendricks-Sturrup | Research Director, Real World Evidence | Duke Margolis Center for Health Policy » speaking at Orphan Drug Congress

Matthew Hewitt, Executive Director, Scientific Services Cell and Gene Therapy, Charles River Laboratories

Matthew Hewitt | Executive Director, Scientific Services Cell and Gene Therapy | Charles River Laboratories » speaking at Orphan Drug Congress

Claudia Hirawat, Owner & Executive Chair, VOZ Advisors

Claudia Hirawat | Owner & Executive Chair | VOZ Advisors » speaking at Orphan Drug Congress

Juliet Hulse, Senior Director, Global RN Strategy and Patient Advocacy, Illingworth Research

Juliet Hulse | Senior Director, Global RN Strategy and Patient Advocacy | Illingworth Research » speaking at Orphan Drug Congress

Gwen Hyland, Head of Commercial Excellence, Rare Diseases, AstraZeneca, Alexion Pharmaceuticals

Gwen Hyland | Head of Commercial Excellence, Rare Diseases, AstraZeneca | Alexion Pharmaceuticals » speaking at Orphan Drug Congress

Hearn Jay Cho, Chief Medical Officer, Multiple Myeloma Research Foundation

Hearn Jay Cho | Chief Medical Officer | Multiple Myeloma Research Foundation » speaking at Orphan Drug Congress

Padmini Jayaraman, Director, Corporate and Business Development, Laekna Therapeuics

Padmini Jayaraman | Director, Corporate and Business Development | Laekna Therapeuics » speaking at Orphan Drug Congress

Chris Jones, Partner, Clinical Research, iTakeControl, a Red Nucleus Company

Chris Jones | Partner, Clinical Research | iTakeControl, a Red Nucleus Company » speaking at Orphan Drug Congress

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

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

Rashida Karmali, Chief Executive Officer, Tactical Therapeutics Inc

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

Vikram Karnani, Executive Vice President, President, International, Horizon Therapeutics

Vikram Karnani | Executive Vice President, President, International | Horizon Therapeutics » speaking at Orphan Drug Congress

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 Drug Congress

Lindsay Kehoe, Project Manager, Clinical Trials Transformation Initiative (CTTI)

Lindsay Kehoe | Project Manager | Clinical Trials Transformation Initiative (CTTI) » speaking at Orphan Drug Congress

Ken Kengatharan, Managing General Partner, Atheneos Ventures

Ken Kengatharan | Managing General Partner | Atheneos Ventures » speaking at Orphan Drug Congress

Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement,, EveryLife Foundation for Rare Diseases

Annie Kennedy | Chief of Policy, Advocacy, & Patient Engagement, | EveryLife Foundation for Rare Diseases » speaking at Orphan Drug Congress

Doug Kerr, Chief Medial Officer, Generation Bio

Doug Kerr | Chief Medial Officer | Generation Bio » speaking at Orphan Drug Congress

Kelly Kester, Director, Advanced Therapy Solutions, Cardinal Health

Kelly Kester | Director, Advanced Therapy Solutions | Cardinal Health » speaking at Orphan Drug Congress

Kritika Khurana, Account Executive, Commercial Team, AllStripes

Kritika Khurana | Account Executive, Commercial Team | AllStripes » speaking at Orphan Drug Congress

Maria Kirsch, General Manager, Patient Services, EVERSANA

Maria Kirsch | General Manager, Patient Services | EVERSANA » speaking at Orphan Drug Congress

Lise Kjems, Chief Medical Officer, Cyclo Therapeutics, Inc.

Lise Kjems | Chief Medical Officer | Cyclo Therapeutics, Inc. » speaking at Orphan Drug Congress

Matthew Klein, Chief Operating Officer, PTC therapeutics

Matthew Klein | Chief Operating Officer | PTC therapeutics » speaking at Orphan Drug Congress

Walter Kowtoniuk, Venture Partner, Third Rock Ventures, LLC

Walter Kowtoniuk | Venture Partner | Third Rock Ventures, LLC » speaking at Orphan Drug Congress

Sarka Kubinova, Medical evaluator, MoCA coordinator, Section of Pricing and Reimbursement Regulation, Czech Republic State Institute for Drug Control

Sarka Kubinova | Medical evaluator, MoCA coordinator, Section of Pricing and Reimbursement Regulation | Czech Republic State Institute for Drug Control » speaking at Orphan Drug Congress

Harsh Kulkarni, Consultant, ZS

Harsh Kulkarni | Consultant | ZS » speaking at Orphan Drug Congress

Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics

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

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

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

David Lapidus, President, Lapidus Data

David Lapidus | President | Lapidus Data » speaking at Orphan Drug Congress

Catherine Lawrence, Head of Rare Disease, Bionical Emas

Catherine Lawrence | Head of Rare Disease | Bionical Emas » speaking at Orphan Drug Congress

Yann Le Cam, Chief Executive Officer, EURORDIS

Yann Le Cam | Chief Executive Officer | EURORDIS » speaking at Orphan Drug Congress

Kimberly LeBlanc, Director, UDN Coordinating Center, Harvard Medical School, Harvard Medical School

Kimberly LeBlanc | Director, UDN Coordinating Center, Harvard Medical School | Harvard Medical School » speaking at Orphan Drug Congress

Seth Lederman, Co-Founder, Chief Executive Officer & Chairman, Tonix Pharmaceuticals

Seth Lederman | Co-Founder, Chief Executive Officer & Chairman | Tonix Pharmaceuticals » speaking at Orphan Drug Congress

Naomi Litchfield, Global Patient Advocacy Lead, Bionical Emas

Naomi Litchfield | Global Patient Advocacy Lead | Bionical Emas » speaking at Orphan Drug Congress

Jeff Livingstone, Chief Executive Officer, Igia Pharmaceuticals

Jeff Livingstone | Chief Executive Officer | Igia Pharmaceuticals » speaking at Orphan Drug Congress

Sam Lucas, SVP- Expanded Access Programs, Durbin- part of Uniphar Group

Sam Lucas | SVP- Expanded Access Programs | Durbin- part of Uniphar Group » speaking at Orphan Drug Congress

Sanjeev Luther, President & Chief Executive Officer, Cornerstone Pharmaceuticals

Sanjeev Luther | President & Chief Executive Officer | Cornerstone Pharmaceuticals » speaking at Orphan Drug Congress

Wolfram Lux, Senior Director, Market Access & Commercial Strategy, Rare Diseases,, Simon-Kucher & Partners

Wolfram Lux | Senior Director, Market Access & Commercial Strategy, Rare Diseases, | Simon-Kucher & Partners » speaking at Orphan Drug Congress

Flaminia Macchia, Executive Director, Rare Diseases International

Flaminia Macchia | Executive Director | Rare Diseases International » speaking at Orphan Drug Congress

Geoff Mackay, President And Chief Executive Officer, AVROBIO

Geoff Mackay | President And Chief Executive Officer | AVROBIO » speaking at Orphan Drug Congress

Natalia Maeva, President, The Bulgarian Society of Patients with Pulmonary Hypertension

Natalia Maeva | President | The Bulgarian Society of Patients with Pulmonary Hypertension » speaking at Orphan Drug Congress

Nitin Mahadev, Vice President, Global Franchise Lead, Rare Disease, Pfizer

Nitin Mahadev | Vice President, Global Franchise Lead, Rare Disease | Pfizer » speaking at Orphan Drug Congress

Will Maier, Vice President, Rare Disease, Drug Development Sciences, ICON plc

Will Maier | Vice President, Rare Disease, Drug Development Sciences | ICON plc » speaking at Orphan Drug Congress

Nick Marchant, Patient & Health Impact lead, Rare Disease, Pfizer

Nick Marchant | Patient & Health Impact lead, Rare Disease | Pfizer » speaking at Orphan Drug Congress

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 Drug Congress

Michael Marquez, Nanoscope Therapeutics

Michael Marquez |  | Nanoscope Therapeutics » speaking at Orphan Drug Congress

Craig Martin, Chief Executive Officer, Global Genes

Craig Martin | Chief Executive Officer | Global Genes » speaking at Orphan Drug Congress

Brad Martin, Vice President, Market Access, Taysha Gene Therapies

Brad Martin | Vice President, Market Access | Taysha Gene Therapies » speaking at Orphan Drug Congress

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

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

Liz Mascherino, Associate Director of Patient Engagement, Equity and Transparency, Biogen

Liz Mascherino | Associate Director of Patient Engagement, Equity and Transparency | Biogen » speaking at Orphan Drug Congress

Carie Masoner, Executive Director, Regulatory & Scientific Affairs, CTI Clinical Trial and Consulting

Carie Masoner | Executive Director, Regulatory & Scientific Affairs | CTI Clinical Trial and Consulting » speaking at Orphan Drug Congress

Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research

Mary McGowan | Chief Executive Officer | Foundation for Sarcoidosis Research » speaking at Orphan Drug Congress

Kelly McKee, Vice President, Patient Registries and Recruitment, Medidata

Kelly McKee | Vice President, Patient Registries and Recruitment | Medidata » speaking at Orphan Drug Congress

Jennifer McNary, Founder, One Rare

Jennifer McNary | Founder | One Rare » speaking at Orphan Drug Congress

Christine Mcsherry, Chief Executive Officer, Casimir Trials

Christine Mcsherry | Chief Executive Officer | Casimir Trials » speaking at Orphan Drug Congress

Wesley Michael, President, Rare Patient Voice, LLC

Wesley Michael | President | Rare Patient Voice, LLC » speaking at Orphan Drug Congress

Christeen Moburg, Vice President Patient Advocacy & Government Relations, Sangamo Therapeutics

Christeen Moburg | Vice President Patient Advocacy & Government Relations | Sangamo Therapeutics » speaking at Orphan Drug Congress

Samarendra Mohanty, Founder-President, Chief Scientific Officer, Nanoscope Therapeutics

Samarendra Mohanty | Founder-President, Chief Scientific Officer | Nanoscope Therapeutics » speaking at Orphan Drug Congress

Mona Moonis, NA Brand Lead, Rare Disease, KKI

Mona Moonis | NA Brand Lead, Rare Disease | KKI » speaking at Orphan Drug Congress

Mark Moore, CEO, GemPharmatech US

Mark Moore | CEO | GemPharmatech US » speaking at Orphan Drug Congress

Maria-Cruz Morillo, Global Therapeutic Operations Lead, Allucent

Maria-Cruz Morillo | Global Therapeutic Operations Lead | Allucent » speaking at Orphan Drug Congress

Christopher Moxham, Chief Scientific Officer, Rarebase, PBC

Christopher Moxham | Chief Scientific Officer | Rarebase, PBC » speaking at Orphan Drug Congress

Andrew Mumford, Managing Director, Initiate

Andrew Mumford | Managing Director | Initiate » speaking at Orphan Drug Congress

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

Michael Murphy | Chief Medical & Scientific Officer | Worldwide Clinical Trials » speaking at Orphan Drug Congress

Joseph Musumeci, President, BluePrint Orphan

Joseph Musumeci | President | BluePrint Orphan » speaking at Orphan Drug Congress

Paul Nash, Senior Director, Simon-Kucher & Partners

Paul Nash | Senior Director | Simon-Kucher & Partners » speaking at Orphan Drug Congress

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

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

Indu Navar, Founder and Chief Executive Officer, EverythingALS

Indu Navar | Founder and Chief Executive Officer | EverythingALS » speaking at Orphan Drug Congress

Amy Nicole Nayar, VP US Patient Advocacy and Government Affairs, Novartis Gene Therapies

Amy Nicole Nayar | VP US Patient Advocacy and Government Affairs | Novartis Gene Therapies » speaking at Orphan Drug Congress

Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD)

Edward Neilan | Chief Medical and Scientific Officer | National Organization for Rare Disorders (NORD) » speaking at Orphan Drug Congress

Robert Neusner, Director, Global Commercial Strategy,, Janssen Pharmaceutical companies of Johnson & Johnson

Robert Neusner | Director, Global Commercial Strategy, | Janssen Pharmaceutical companies of Johnson & Johnson » speaking at Orphan Drug Congress

Amanda Nickles Fader, Professor of Gynecology and Obstetrics, Professor of Oncology, Vice-Chair, Gynecologic Surgical Operations,, Johns Hopkins Medical Center

Amanda Nickles Fader | Professor of Gynecology and Obstetrics, Professor of Oncology, Vice-Chair, Gynecologic Surgical Operations, | Johns Hopkins Medical Center » speaking at Orphan Drug Congress

Kris O’Brien, Executive Director, Program Strategy, Rare Disease and Pediatrics, Premier Research

Kris O’Brien | Executive Director, Program Strategy, Rare Disease and Pediatrics | Premier Research » speaking at Orphan Drug Congress

Andrew Obenshain, Chief Executive Officer, Bluebird Bio

Andrew Obenshain | Chief Executive Officer | Bluebird Bio » speaking at Orphan Drug Congress

Matan Ofir, Director, Medison Ventures

Matan Ofir | Director | Medison Ventures » speaking at Orphan Drug Congress

Cristol O'Loughlin, Founder & Chief Executive Officer, ANGEL AID CARES

Cristol O'Loughlin | Founder & Chief Executive Officer | ANGEL AID CARES » speaking at Orphan Drug Congress

Asif Paker, VP Clinical Development, SwanBio Therapeutics

Asif Paker | VP Clinical Development | SwanBio Therapeutics » speaking at Orphan Drug Congress

Jim Palma, Executive Director, TargetCancer Foundation

Jim Palma | Executive Director | TargetCancer Foundation » speaking at Orphan Drug Congress

Francis Pang, VP Global Market Access, Orchard Therapeutics

Francis Pang | VP Global Market Access | Orchard Therapeutics » speaking at Orphan Drug Congress

Lisa Parker, VP, Medical, emotive

Lisa Parker | VP, Medical | emotive » speaking at Orphan Drug Congress

Kinnari Patel, President & COO, Rocket Pharma

Kinnari Patel | President & COO | Rocket Pharma » speaking at Orphan Drug Congress

Bhavesh Patel, Principal, CRA, Charles River Associates

Bhavesh Patel | Principal | CRA, Charles River Associates » speaking at Orphan Drug Congress

Luciana Peixoto, Member of Parents Advisory Board, Systemic JIA Foundation

Luciana Peixoto | Member of Parents Advisory Board | Systemic JIA Foundation » speaking at Orphan Drug Congress

Melissa Penn, Director Patient Engagement Research & Development, Bayer

Melissa Penn | Director Patient Engagement Research & Development | Bayer » speaking at Orphan Drug Congress

Maria Picone, CEO, TREND Community

Maria Picone | CEO | TREND Community » speaking at Orphan Drug Congress

Richa Poddar, Chief Commercial Officer, Agios Pharmaceuticals

Richa Poddar | Chief Commercial Officer | Agios Pharmaceuticals » speaking at Orphan Drug Congress

Ana Puhl, Senior Research Scientist, Collaborations Pharmaceuticals

Ana Puhl | Senior Research Scientist | Collaborations Pharmaceuticals » speaking at Orphan Drug Congress

Pam Rattananont, Director, Customer Strategy and Innovation, Parexel

Pam Rattananont | Director, Customer Strategy and Innovation | Parexel » speaking at Orphan Drug Congress

Shannon Resetich, Global Franchise Head, Rare Diseases, Sanofi Genzyme

Shannon Resetich | Global Franchise Head, Rare Diseases | Sanofi Genzyme » speaking at Orphan Drug Congress

Michael Rice, Senior VP, Head of Advanced Therapeutics, Lumanity

Michael Rice | Senior VP, Head of Advanced Therapeutics | Lumanity » speaking at Orphan Drug Congress

David Ringger, Head of Market Access, Initiate

David Ringger | Head of Market Access | Initiate » speaking at Orphan Drug Congress

David Rose, Business Development, Rare Revolution

David Rose | Business Development | Rare Revolution » speaking at Orphan Drug Congress

Luke Rosen, Founder, KIF1A.ORG

Luke Rosen | Founder | KIF1A.ORG » speaking at Orphan Drug Congress

Johanna Rossell, Chief Commercial Officer, Enzyvant

Johanna Rossell | Chief Commercial Officer | Enzyvant » speaking at Orphan Drug Congress

Ruth Rostron, Director of Program Management, Durbin- part of Uniphar Group

Ruth Rostron | Director of Program Management | Durbin- part of Uniphar Group » speaking at Orphan Drug Congress

Christopher Rudolf, CEO, volv global

Christopher Rudolf | CEO | volv global » speaking at Orphan Drug Congress

Ashish Sagrolikar, Chief Operating Officer, Zogenix Inc.

Ashish Sagrolikar | Chief Operating Officer | Zogenix Inc. » speaking at Orphan Drug Congress

Dharmendra Sahay, ZS

Dharmendra Sahay |  | ZS » speaking at Orphan Drug Congress

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 Drug Congress

Denise Schulz, Associate Director of Clinical Operations,, Horizon Therapeutics

Denise Schulz | Associate Director of Clinical Operations, | Horizon Therapeutics » speaking at Orphan Drug Congress

Simone Seiter, Partner, Simon-Kucher & Partners

Simone Seiter | Partner | Simon-Kucher & Partners » speaking at Orphan Drug Congress

Antonino Severino, Head CTS, Nextpharma

Antonino Severino | Head CTS | Nextpharma » speaking at Orphan Drug Congress

Gopi Shanker, Chief Scientific Officer, Tevard Biosciences

Gopi Shanker | Chief Scientific Officer | Tevard Biosciences » speaking at Orphan Drug Congress

Panna Sharma, Chief Executive Officer, President and Director, Lantern Pharma

Panna Sharma | Chief Executive Officer, President and Director | Lantern Pharma » speaking at Orphan Drug Congress

Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research

Tricha Shivas | Chief Strategy Officer | Foundation for Sarcoidosis Research » speaking at Orphan Drug Congress

Fanny Sie, One Roche, Head of Artificial Intelligence and Digital Health, Roche

Fanny Sie | One Roche, Head of Artificial Intelligence and Digital Health | Roche » speaking at Orphan Drug Congress

Stuart Siedman, Head of Patient Advocacy, Chiesi Global Rare Disease

Stuart Siedman | Head of Patient Advocacy | Chiesi Global Rare Disease » speaking at Orphan Drug Congress

Amy Simon, Chief Medical Officer, Beam Therapeutics

Amy Simon | Chief Medical Officer | Beam Therapeutics » speaking at Orphan Drug Congress

Jaswinder Singh Khera, Managing Director, WEP Clinical

Jaswinder Singh Khera | Managing Director | WEP Clinical » speaking at Orphan Drug Congress

Nick Sireau, Chair and Chief Executive Officer, AKU Society

Nick Sireau | Chair and Chief Executive Officer | AKU Society » speaking at Orphan Drug Congress

Jayson Slotnik, Partner, Health Policy Strategies, LLC

Jayson Slotnik | Partner | Health Policy Strategies, LLC » speaking at Orphan Drug Congress

Charlie Small, Associate Director, Integrated Trial Solutions, Elligo Health Research

Charlie Small | Associate Director, Integrated Trial Solutions | Elligo Health Research » speaking at Orphan Drug Congress

Todd Smith, President, Chief Commercial Officer, Tolmar Pharmaceuticals Inc

Todd Smith | President, Chief Commercial Officer | Tolmar Pharmaceuticals Inc » speaking at Orphan Drug Congress

Scott Snyder, Chief Digital Officer, EVERSANA

Scott Snyder | Chief Digital Officer | EVERSANA » speaking at Orphan Drug Congress

Charlene Son Rigby, Chief Executive Officer, RARE-X

Charlene Son Rigby | Chief Executive Officer | RARE-X » speaking at Orphan Drug Congress

Matt Sowards, Chief Innovation Officer, Scout Clinical

Matt Sowards | Chief Innovation Officer | Scout Clinical » speaking at Orphan Drug Congress

Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products, Medicines Evaluation Board, Medicines Evaluation Board

Violeta Stoyanova-Beninska | Chair of Committee for Orphan Medicinal Products, Medicines Evaluation Board | Medicines Evaluation Board » speaking at Orphan Drug Congress

Swami Subramanian, Head, US/Global Pricing and Contracting, Regeneron

Swami Subramanian | Head, US/Global Pricing and Contracting | Regeneron » speaking at Orphan Drug Congress

Dean Suhr, President, MLD Foundation

Dean Suhr | President | MLD Foundation » speaking at Orphan Drug Congress

Jamie Sullivan, Director of Public Policy, EveryLife Foundation

Jamie Sullivan | Director of Public Policy | EveryLife Foundation » speaking at Orphan Drug Congress

Noreen Sullivan, Senior Principal, Value & Development Consulting, Evidera PPD

Noreen Sullivan | Senior Principal, Value & Development Consulting | Evidera PPD » speaking at Orphan Drug Congress

Web Sun, Co-Founder & President, Komodo Health

Web Sun | Co-Founder & President | Komodo Health » speaking at Orphan Drug Congress

Lee Sutton, Vice President, Business Development, WEP Clinical

Lee Sutton | Vice President, Business Development | WEP Clinical » speaking at Orphan Drug Congress

Tim Szymusiak, Head of Market Access Services, BluePrint Orphan

Tim Szymusiak | Head of Market Access Services | BluePrint Orphan » speaking at Orphan Drug Congress

Mitra Tavakkoli, VP, Clinical Development, -

Mitra Tavakkoli | VP, Clinical Development | - » speaking at Orphan Drug Congress

Amy Teets, Global Public Affairs Head, Pompe and MPS, Sanofi Genzyme

Amy Teets | Global Public Affairs Head, Pompe and MPS | Sanofi Genzyme » speaking at Orphan Drug Congress

Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences

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

Michael Thomas, Principal, ZS

Michael Thomas | Principal | ZS » speaking at Orphan Drug Congress

Kate Tighe, Head, US Public Affairs & Patient Advocacy - Rare Diseases, Sanofi Genzyme

Kate Tighe | Head, US Public Affairs & Patient Advocacy - Rare Diseases | Sanofi Genzyme » speaking at Orphan Drug Congress

Mark Trusheim, Strategic Director, MIT NEWDIGS

Mark Trusheim | Strategic Director | MIT NEWDIGS » speaking at Orphan Drug Congress

Andrew Udell, President of North America, Commercial, Calliditas Therapeutics

Andrew Udell | President of North America, Commercial | Calliditas Therapeutics » speaking at Orphan Drug Congress

Fyodor Urnov, Scientific Director, Innovative Genomics Institute, Professor, Department of Molecular & Cell Biology, University of California, Berkeley

Fyodor Urnov | Scientific Director, Innovative Genomics Institute, Professor, Department of Molecular & Cell Biology | University of California, Berkeley » speaking at Orphan Drug Congress

Zizi Uzezi Imatorbhebhe, Senior Vice President, Global Strategy and Development, Head Rare Disease Innovation Center,, Ergomed

Zizi Uzezi Imatorbhebhe | Senior Vice President, Global Strategy and Development, Head Rare Disease Innovation Center, | Ergomed » speaking at Orphan Drug Congress

Henri Vaitkevicius, VP of Clinical Development,, Marinus Pharmaceuticals

Henri Vaitkevicius | VP of Clinical Development, | Marinus Pharmaceuticals » speaking at Orphan Drug Congress

Kristin Van Goor, Lead, North America Regulatory Policy, Vertex Pharmaceuticals

Kristin Van Goor | Lead, North America Regulatory Policy | Vertex Pharmaceuticals » speaking at Orphan Drug Congress

Suneet Varma, Global President, Rare Disease Business Unit, Pfizer inc

Suneet Varma | Global President, Rare Disease Business Unit | Pfizer inc » speaking at Orphan Drug Congress

Geeta Vemuri, Managing Partner, Agent Capital

Geeta Vemuri | Managing Partner | Agent Capital » speaking at Orphan Drug Congress

Richard Vesely, Vice President, Regulatory Strategy,, Allucent

Richard Vesely | Vice President, Regulatory Strategy, | Allucent » speaking at Orphan Drug Congress

Erin Wade, Content Specialist, myTomorrows

Erin Wade | Content Specialist | myTomorrows » speaking at Orphan Drug Congress

Mary Wang, Science Policy Manager, Rare Diseases International

Mary Wang | Science Policy Manager | Rare Diseases International » speaking at Orphan Drug Congress

Dan Wasserstrom, Senior Director, Business Development, Clinigen Group

Dan Wasserstrom | Senior Director, Business Development | Clinigen Group » speaking at Orphan Drug Congress

Christopher Watson, Director, Consulting, Thread Research

Christopher Watson | Director, Consulting | Thread Research » speaking at Orphan Drug Congress

Kevin Weatherwax, Vice President, Head of Regulatory Affairs, WEP Clinical

Kevin Weatherwax | Vice President, Head of Regulatory Affairs | WEP Clinical » speaking at Orphan Drug Congress

Robert Weinstein, Division of Infectious Diseases, Rush University

Robert Weinstein | Division of Infectious Diseases | Rush University » speaking at Orphan Drug Congress

Ellen Weiss, Vice President, In-Home Solutions, Decentralized Clinical Trials, PCM Trials

Ellen Weiss | Vice President, In-Home Solutions, Decentralized Clinical Trials | PCM Trials » speaking at Orphan Drug Congress

Gareth Williams, President - Sciensus Rare, Sciensus

Gareth Williams | President - Sciensus Rare | Sciensus » speaking at Orphan Drug Congress

Toni Williams, Global Business Development Director, Clinigen Group

Toni Williams | Global Business Development Director | Clinigen Group » speaking at Orphan Drug Congress

Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Disorders

Durhane Wong-Rieger | President & Chief Executive Officer | Canadian Organization for Rare Disorders » speaking at Orphan Drug Congress

Richard Xie, Health Economist & Director of Research, The Innovation and Value Initiative IVI

Richard Xie | Health Economist & Director of Research | The Innovation and Value Initiative IVI » speaking at Orphan Drug Congress

Rachel Yang, Secretary, Council Member, RDI, Head of International Affairs, Chinese Alliance for Rare Diseases (CHARD)

Rachel Yang | Secretary, Council Member, RDI, Head of International Affairs | Chinese Alliance for Rare Diseases (CHARD) » speaking at Orphan Drug Congress

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

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

Christine Yu, Chief Executive Officer, P2P Syncro

Christine Yu | Chief Executive Officer | P2P Syncro » speaking at Orphan Drug Congress

Nancy Yu, Co-Founder & Chief Executive Officer, AllStripes Research

Nancy Yu | Co-Founder & Chief Executive Officer | AllStripes Research » speaking at Orphan Drug Congress

Martine Zimmermann, Global Head Of Regulatory Affairs, Alexion Pharmaceuticals

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

Shiran Zimri, Head of Scientific Program, NeuroSense Therapeutics

Shiran Zimri | Head of Scientific Program | NeuroSense Therapeutics » speaking at Orphan Drug Congress

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