2024 Speakers

 

Suchismita Acharya, Chief Executive Officer, AyuVis Research

Suchismita Acharya | Chief Executive Officer | AyuVis Research » speaking at Orphan Drug Congress

Samuel Agyei Wiafe, Executive Director, Rare Disease Ghana Initiative

Dennis Akkaya, Chief Commercial Officer, myTomorrows

David Allison, Chief Executive Officer, TREAT-NMD

David Allison | Chief Executive Officer | TREAT-NMD » speaking at Orphan Drug Congress

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

Irina Antonijevic, Chief Medical Officer, EveryONE Medicines

Irina Antonijevic | Chief Medical Officer | EveryONE Medicines » speaking at Orphan Drug Congress

Victoria Arteaga, Co-founder, SHER Hispanic Society for Rare Diseases

Victoria Arteaga | Co-founder | SHER Hispanic Society for Rare Diseases » speaking at Orphan Drug Congress

Ethan Ash, Senior VP of Business Development, BioNews

Ethan Ash | Senior VP of Business Development | BioNews » speaking at Orphan Drug Congress

Kevin Bagley, Former Medicaid Director for State of Nebraska | Consultant, Kevin Bagley Strategic Consulting

Mark Bainbridge, Senior Director, Business Development, Early Access, Inceptua Group

Ritu Baral, Managing Director And Senior Biotechnology Analyst, Cowen

Ritu Baral | Managing Director And Senior Biotechnology Analyst | Cowen » speaking at Orphan Drug Congress

Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance

Ron Bartek | President, Co-Founder | Friedreich's Ataxia Research Alliance » speaking at Orphan Drug Congress

Kevin Baruzzi, Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH

Stuart Bell, Senior Vice President, Inceptua Early Access, Inceptua Group

Jana Benesh, Director of Therapeutic Expertise, ICON Centre for Rare Diseases

Kara Berasi, Chief Executive Officer, Haystack Project

Alexandre Bétourné, Executive Director, Critical Path Institute

Sulagna Bhattacharya, Chief Executive Officer, Nanoscope Therapeutics

Robin Bliss, VP Strategic Consultant, Veristat

Lara Bloom, President and CEO, The Ehlers-Danlos Society

Adam Bloomfield, M.D., FAAP, Executive Director, Medical Affairs, Rare Diseases and Pediatrics, Premier Research

Morgan Boname, Senior Director, Public Policy, Neurocrine Biosciences

Natasha Bonhomme, Founder, Expecting Health

James Boomhower MS(c), FP-C, NR-P, CCISM, Code Rare EMS Educator, Rare STRIDES

Wendy Borsari, Sr. Manager of Patient Advocacy, Tenaya Therapeutics

Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

P.J. BROOKS, Deputy Director of the NCATS Division of Rare Diseases Research Innovation, National Institutes of Health

Benjamin Brown, Executive Director, American Society of Pharmacovigilance

Benjamin Brown | Executive Director | American Society of Pharmacovigilance » speaking at Orphan Drug Congress

Bert Bruce, US Rare Disease Lead, Pfizer, Pfizer

Chuck Bucklar, Principal, Chair 5 Consulting, LLC

Karin Butler, Head of Technical Research, York Health Economics Consortium

Lauren Bylsma, Senior Epidemiologist, EpidStrategies, A Division of ToxStrategies

Lauren Bylsma | Senior Epidemiologist | EpidStrategies, A Division of ToxStrategies » speaking at Orphan Drug Congress

Amanda Cali, Trustee,The Radiant Hope Foundation; Trustee,Ian Cali FOP Research Fund at University of PA; Co-Founder/Officer, Tin Soldiers Global

Amanda Cali | Trustee,The Radiant Hope Foundation; Trustee,Ian Cali FOP Research Fund at University of PA; Co-Founder/Officer | Tin Soldiers Global » speaking at Orphan Drug Congress

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

Joshua Card-Gowers, Senior Evidence Lead, HealthLumen

Madeleine Carrier, Patient Navigator, myTomorrows

Madeleine Carrier | Patient Navigator | myTomorrows » speaking at Orphan Drug Congress

Geoffrey Case, Digital Editor, RARE Revolution Magazine

Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience

Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics

Grant Castor | SVP Commercial Strategy & Operations | Sentynl Therapeutics » speaking at Orphan Drug Congress

Chelsea Catsburg, Engagement Manager, BluePrint Orphan

Chelsea Catsburg | Engagement Manager | BluePrint Orphan » speaking at Orphan Drug Congress

Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi

Anne-Sophie Chalandon | Head of Global Public Affairs, Rare Diseases and CGT Policies | Sanofi » speaking at Orphan Drug Congress

James Chambers, Associate Professor, Tufts Medical Center

Chere Chapman, Chief Executive Officer, Ardea Outcomes

Richard Chapman, Chief Science Officer, Innovation and Value Initiative

Richard Chapman | Chief Science Officer | Innovation and Value Initiative » speaking at Orphan Drug Congress

Ellen Cho, Director, Specialty Programs, Direct Relief

Priya S Chockalingam, Vice President, Head of Clinical Bioanalytics & Translational Sciences, Beam Therapeutics

Duane Clark, General Manager, US Rare Disease, Sanofi

Kyle Clifton, Senior Director, Red Nucleus

Jason Colquitt, Chief Executive Officer, Across Healthcare

Matt Conway, Vice President of Strategic Initiatives, Rare STRIDES

Colin Cook, Technical Founder, XDemics Corporation

Colin Cook | Technical Founder | XDemics Corporation » speaking at Orphan Drug Congress

Ron Cooper, Former CEO, Albireo Pharma

Donna Cowan, Associate Director EAP, Stealth BioTherapeutics

Carole Cramer, President & Founder, BioStrategies

Joslyn Crowe, Executive Director, National Niemann Pick Disease Foundation Inc

Joslyn Crowe | Executive Director | National Niemann Pick Disease Foundation Inc » speaking at Orphan Drug Congress

Andrew Cummins, VP Access and Reimbursement, Sciensus

Colleen Dansereau, Senior Director - Gene Therapy Program and Office of Emerging Medical Discoveries, Boston Children's Hospital

Michelle Davis, Executive Director, International Fibrodysplasia Ossificans Progressiva (FOP) Association, Inc.

Khrystal K Davis, Founding President, Texas Rare Alliance

Khrystal K Davis | Founding President | Texas Rare Alliance » speaking at Orphan Drug Congress

Marcella Debidda, President, BioNews Clinical, BioNews Clinical

Daniel DeFabio, Rare Dad (Menkes Disease), Co-Founder, Disorder: The Rare Disease Film Festival

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

Linda Dell, VP, Program Leader, ReAlta Life Sciences, Inc.

Jin Ding, Research Fellow, University of Sheffield

Kate Donigan, Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs, Sarepta Therapeutics

Kate Donigan | Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs | Sarepta Therapeutics » speaking at Orphan Drug Congress

Lauren Dougherty, Chief Operating Officer, TREND Community

Kasey Edwards, Co-Founder, Community Relations Specialist, Cure AP-4

Kasey Edwards | Co-Founder, Community Relations Specialist | Cure AP-4 » speaking at Orphan Drug Congress

Leslie Edwin, President, Cushing's Support & Research Foundation

Michael Eging, Executive Director, Rare Access Action Project

Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals

Jida El Hajjar, Executive Director,, ALS Action Canada

Jida El Hajjar | Executive Director, | ALS Action Canada » speaking at Orphan Drug Congress

Shaun Ellis, Vice President, Global Market Access Specialty Ophthalmology, Johnson & Johnson

Terri Ellsworth, Patient Advocate Partnerships, myTomorrows

Terri Ellsworth | Patient Advocate Partnerships | myTomorrows » speaking at Orphan Drug Congress

Bastiaan Evers, Senior Director Drug Discovery, Scenic Biotech

Bastiaan Evers | Senior Director Drug Discovery | Scenic Biotech » speaking at Orphan Drug Congress

Erik Feingold, CEO & Co-Founder, FDNA, Inc

James Fink, Director, Epilepsy Therapeutic Lead, Quiver Bioscience

Alicia Fiscus, Head of Global Regulatory Affairs and QA, Rhythm Pharmaceuticals

Colton Flowers, NLP Engineer, TREND Community

Colton Flowers | NLP Engineer | TREND Community » speaking at Orphan Drug Congress

Pawel Fludzinski, Chief Executive Officer, AmideBio

Max Flurie, Senior Data Scientist, TREND Community

Max Flurie | Senior Data Scientist | TREND Community » speaking at Orphan Drug Congress

Benjamin Forred, Director of Translational Research and the CoRDS Registry, Sanford Health

Lindy Forte, Senior Vice President, Market Access and Pricing, EVERSANA

Jean-Pascal Fournier-Roussy, Head, Global Public Affairs, Rare Diseases & Rare Blood Disorders, Sanofi

Nick France, Vice President, Clinical Development, Neurology, Alexion, AstraZeneca Rare Disease

Nick France | Vice President, Clinical Development, Neurology | Alexion, AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Sebastian Franzinger, Head of Business Development, Scenic Biotech

Albert Freedman, Counseling Psychologist / Rare Dad, Rarecounseling.com

Albert Freedman | Counseling Psychologist / Rare Dad | Rarecounseling.com » speaking at Orphan Drug Congress

Jon Fryzek, Practice Director, EpidStrategies, A Division of ToxStrategies

Jon Fryzek | Practice Director | EpidStrategies, A Division of ToxStrategies » speaking at Orphan Drug Congress

Mike Fusakio, Lead Consultant, Regulatory Affairs, Halloran Consulting Group

Renata Gallagher, MD, PhD, Medical Director, Parexel

Neha Gandhi, Consultant, Advocacy and Policy

Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)

Pamela Gavin | Executive Vice President | National Organization for Rare Disorders (NORD) » speaking at Orphan Drug Congress

Sarah Gheuens, Chief Medical Officer, Head of R&D, Agios Pharmaceuticals

Sarah Gheuens | Chief Medical Officer, Head of R&D | Agios Pharmaceuticals » speaking at Orphan Drug Congress

Diego Gil Cardozo, President, Enfermedades Raras en el Caribe y América Latina (ERCAL)

Diego Gil Cardozo | President | Enfermedades Raras en el Caribe y América Latina (ERCAL) » speaking at Orphan Drug Congress

Christoph Glaetzer, Chief Global Value and Access Officer, Johnson & Johnson

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

Sarah Glass, Chief Operating Officer, n-Lorem Foundation

Josie Godfrey, Co-Founder and Chief Executive Officer, Realise Advocacy

Josie Godfrey | Co-Founder and Chief Executive Officer | Realise Advocacy » speaking at Orphan Drug Congress

Leslie Gordon, Medical Director, Progeria Research Foundation

Deb Gouveia, SVP, Development Operations, EveryONE Medicines

Heidi Grabenstatter, Science Director, Critical Path Institute

Alicia Granados, Head, Global Rare Diseases Medical Scientific Advocacy, SANOFI

Nathan Grant, Researcher, Medical Student; President and Founder, Harvard Medical School; Siblings with a Mission

Amy Gray, CEO, Undiagnosed Diseases Network Foundation

Brandi Greenberg, VP Marketing-Analytics & Insights, Real Chemistry

James Griffin, Sickle Cell Patient Advocate, Independent Milwaukee Wisconsin

Gay Grossman, Co-Founder and Patient Advocate, ADCY5.org

Teri Hagemann, Director of Client Services, Splash Clinical

Alaa Hamed, Global Head, Medical Affairs Rare Disease and Rare Blood Disorders, Sanofi

Alaa Hamed | Global Head, Medical Affairs Rare Disease and Rare Blood Disorders | Sanofi » speaking at Orphan Drug Congress

Matthew Hanchard, Research Fellow, University of Sheffield

Kate Hanman, US Head of Rare Diseases, Costello Medical

Rachel Harrison, Pre-Approval Access Lead, Argenx

Rachel Harrison | Pre-Approval Access Lead | Argenx » speaking at Orphan Drug Congress

Sue Harvey, Founder & Chief Transformation Officer, Riverwise Leadership

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

Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International

Gary Ho, Senior Community Director, TREND Community

Laura Hopkins, Associate Director, Critical Path Institute

Laura Hopkins | Associate Director | Critical Path Institute » speaking at Orphan Drug Congress

Spencer Huggett, Founder, Synapze

Spencer Huggett | Founder | Synapze » speaking at Orphan Drug Congress

Wills Hughes-Wilson, Chief Patient Access & Commercial Planning, Mereo Biopharma

Afshan Hussain, Managing Director, Global Rare Disease Lead, Havas Health

Afshan Hussain | Managing Director, Global Rare Disease Lead | Havas Health » speaking at Orphan Drug Congress

Huong Huynh, Director of Regulatory Science, Critical Path Institute

Oxana Iliach, Member of Regulatory Science Committee, International Rare Diseases Research Consortium (IRDiRC)

Katheron Intson, Chief Executive Officer, Varient

Roberto Iunes, Senior Health Economist, World Bank

Roberto Iunes | Senior Health Economist | World Bank » speaking at Orphan Drug Congress

Sun-Gou Ji, Vice President of Computational Genetics, BridgeBio Pharma

Gretchen Jordan, Associate Director, Cushing's Support & Research Foundation

Andria Joseph, Project Director Outcomes Research, York Health Economics Consortium

Christine Juday, Head, Market Access, Real Chemistry

Richie Kahn, Co-Founder & Principal, Canary Advisors

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

Inderpreet Kambo, Principal (Partner) - Information Management & Analytics, IQVIA

Manohar Katakam, Chief Executive Officer, SteroTherapeutics

Ken Kengatharan, Managing General Partner, Atheneos Ventures

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

Melissa Kennedy, Chief Executive Officer, International Rett Syndrome Foundation

Melissa Kennedy | Chief Executive Officer | International Rett Syndrome Foundation » speaking at Orphan Drug Congress

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

Adrian Kielhorn, Senior Director Health Economics and Outcomes Research, Alexion

Amy Kimzey, Toxicologist, EpidStrategies, A Division of ToxStrategies

Amy Kimzey | Toxicologist | EpidStrategies, A Division of ToxStrategies » speaking at Orphan Drug Congress

MARIA KIRSCH, President, Patient Services, EVERSANA

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

Terri Klein | President & Chief Executive Officer | National M.P.S. Society » speaking at Orphan Drug Congress

Aki Ko, Chief Executive Officer, Elixirgen Therapeutics, Inc.

Bret Koncak, Co-Founder, mejo

Lauren Kopsick, Founder and Executive Director, The Healthcare Navigation Project

Augusta Elizabeth Koroma, Chief Executive Officer, Sickle cell Intervention U.K

Augusta Elizabeth Koroma | Chief Executive Officer | Sickle cell Intervention U.K » speaking at Orphan Drug Congress

Ivan Kouchlev, Managing Principal, Real-World Data Solutions, Real Chemistry

Kollet Koulianos, Senior Payor Advisor, National Hemophilia Foundation

Kollet Koulianos | Senior Payor Advisor | National Hemophilia Foundation » speaking at Orphan Drug Congress

Rahul Kukreja, Project Director, Clinical Project Management, Allucent

Neha Kumar, Global Commercial Strategy - Precision Medicine, Johnson & Johnson Innovative Medicine

Alix Lacoste, AI Computational Biology Director Genetic Insights, Invitae

Alix Lacoste | AI Computational Biology Director Genetic Insights | Invitae » speaking at Orphan Drug Congress

Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen

Shira Landskroner-Eiger | Director, External Innovation Rare Disease | Ipsen » speaking at Orphan Drug Congress

Larissa Lapteva, Associate Director, FDA

Larissa Lapteva | Associate Director | FDA » speaking at Orphan Drug Congress

Sherry Lard, PhD, Associate Director for Product Management (ADPM), U.S. Food and Drug Administration

Patroski Lawson, Chief Executive Officer, The KPM Group DC

Patroski Lawson | Chief Executive Officer | The KPM Group DC » speaking at Orphan Drug Congress

Yann Le Cam, Founder and past-CEO, EURORDIS

Yann Le Cam | Founder and past-CEO | EURORDIS » speaking at Orphan Drug Congress

Connie Lee, President and Chief Executive Officer, Alliance to Cure Cavernous Malformations

Michael Lehmicke, Senior Vice President of Science and Industry Affairs, Alliance for Regenerative Medicine

Michael Lehmicke | Senior Vice President of Science and Industry Affairs | Alliance for Regenerative Medicine » speaking at Orphan Drug Congress

Melanie Lendnal, Senior Vice President, Policy and Advocacy, The ALS Association

Melanie Lendnal | Senior Vice President, Policy and Advocacy | The ALS Association » speaking at Orphan Drug Congress

Maurice Leonard, Medical Director, Uniphar

William Lin, Program Director, GARDaccess, Partnership for Quality Medical Donations (PQMD)

William Lin | Program Director, GARDaccess | Partnership for Quality Medical Donations (PQMD) » speaking at Orphan Drug Congress

Andrew Lo, Charles E. and Susan T. Harris Professor, Massachusetts Institute of Technology (MIT)

Robert Long, Executive Director, Uplifting Athletes

Robert Long | Executive Director | Uplifting Athletes » speaking at Orphan Drug Congress

Veronica Lopez Gousset, MPH, Founder and President, VLG Global

Cathleen Lutz, Vice President of the Rare Disease Translational Center, Jackson Laboratory

Gary M Hutchinson, SVP, Cold Chain Engineering, Modality Solutions

Kim MacDonnell, Associate Director, Rare Disease, COE, Parexel

Kim MacDonnell | Associate Director, Rare Disease, COE | Parexel » speaking at Orphan Drug Congress

Donna Mackey, Vice President, Clinical Operations, Akouos, Inc.

Donna Mackey | Vice President, Clinical Operations | Akouos, Inc. » speaking at Orphan Drug Congress

Mark Manfredi, Chief Executive Officer, Ikena Oncology

Mark Manfredi | Chief Executive Officer | Ikena Oncology » 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

Craig Martin, President, Rithm Health; CEO, Orphan Therapeutics Accelerator

Jaclyn Martin, Vice President of Global Head Market Access Cell and Gene Therapy, Bayer

Silas Martin, Head, Access & Policy Research, Johnson & Johnson

Silas Martin | Head, Access & Policy Research | Johnson & Johnson » speaking at Orphan Drug Congress

Kristin Marvin-Keller, Managing Director, Havas Life Rare

Kristin Marvin-Keller | Managing Director | Havas Life Rare » speaking at Orphan Drug Congress

SWEETY MATHEW, Global CMC Product Lead- CMC Biotech & Rare Diseases, Novo Nordisk

Annette Maughan, CEO/Co-Founder, KBG Foundation

Reenie McCarthy, Chief Executive Officer, Stealth BioTherapeutics

Reenie McCarthy | Chief Executive Officer | Stealth BioTherapeutics » speaking at Orphan Drug Congress

Alice McConnell, Chief Executive Officer, Speragen, Inc

Michael McGinn, Founder, CureLBSL

Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research

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

Gina McHugh, Conference Producer, Terrapinn

Gina McHugh | Conference Producer | Terrapinn » speaking at Orphan Drug Congress

Kristin McKay, President and Executive Director, Project Alive

Jennifer McNary, Founder, One Rare

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

Stuart Mealing, Associate Director (HEOR), York Health Economics Consortium

Laurence Mignon, Executive Director, Clinical Development, n-Lorem Foundation

Sheila Mikhail, Executive Director, Columbus Children's Foundation

Sheila Mikhail | Executive Director | Columbus Children's Foundation » speaking at Orphan Drug Congress

Tsveta Milanova, Chief Commercial Officer, Agios Pharmaceuticals

Tsveta Milanova | Chief Commercial Officer | Agios Pharmaceuticals » speaking at Orphan Drug Congress

Nicola Miller, Editor in Chief, RARE Revolution Magazine

Nicola Miller | Editor in Chief | RARE Revolution Magazine » speaking at Orphan Drug Congress

Nicole Miller, Vice President, Molecular Diagnostics, Global Medical Affairs, Ultragenyx

Nicole Miller | Vice President, Molecular Diagnostics, Global Medical Affairs | Ultragenyx » speaking at Orphan Drug Congress

Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease, Worldwide Clinical Trials

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

James Mongiardo, Chief Executive Officer, A-Synaptic Corp.

Mary Morlino, Patient Navigator, Undiagnosed Disease Network Foundation (UDNF)

Lindsay Murphy, Senior Group Director, Integrated Intelligence, Real Chemistry

Joe Musumeci, President, BluePrint Orphan

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

Ramaiah Muthyala, Professor/Director at University of Minnesota, President and Chief Executive Officer,, Indian organization for Rare diseases

Ramaa Nathan, VP of Data Science and Real-World Evidence, EVERSANA

Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs

Alexander Natz | Director General | EUCOPE - European Confederation of Pharmaceutical Entrepreneurs » speaking at Orphan Drug Congress

Neena Nizar, Assoc. Director, Patient Advocacy Strategy, ICON Centre for Rare Diseases; President, The Jansens Foundation

Greg Palko, Vice President, North America Oncology Region Franchise Head, Kyowa Kirin

Jim Palma, Chief Executive Officer, TargetCancer Foundation

Effie Parks, Podcast Host, Director of Parnerships, Once Upon A Gene - CTNNB1

Kinnari Patel, President & COO, Rocket Pharma

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

Dave Pearce, Chair, International Rare Diseases Research Consortium (IRDiRC)

Melissa Penn, Director Patient Engagement Research & Development, Bayer

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

Sue Peschin, President and Chief Executive Officer, Alliance for Aging Research

Steven Peskin, President and Chief Executive Officer; Former Executive Medical Director Population Health, Horizon Blue Cross Blue Shield of NJ, SRP Advisors, LLC

Steven Peskin | President and Chief Executive Officer; Former Executive Medical Director Population Health, Horizon Blue Cross Blue Shield of NJ | SRP Advisors, LLC » speaking at Orphan Drug Congress

Maria Picone, Chief Executive Officer, TREND Community

Maria Picone | Chief Executive Officer | TREND Community » speaking at Orphan Drug Congress

Peter Pitts, President, C.M.P.I

Christopher Porter, Vice President, Government Affairs and Policy, Travere Therapeutics

Christopher Porter | Vice President, Government Affairs and Policy | Travere Therapeutics » speaking at Orphan Drug Congress

Otto Prohaska, Founder and Chief Executive Officer, Transcytos

XENIA PROTON DE LA CHAPELLE, Chief Executive Officer and Patient Representative, AtmosR

Harsha Rajasimha, Founder and Chairman, IndoUSrare

Harpreet Ram, President, EVR Consulting

Harpreet Ram | President | EVR Consulting » speaking at Orphan Drug Congress

Dorian Readnour, Executive Director, Global Patient Access, Sarepta Therapeutics

Dorian Readnour | Executive Director, Global Patient Access | Sarepta Therapeutics » speaking at Orphan Drug Congress

Debra Regier, Director, Rare Disease Institute, Children's National Hospital

Debra Regier | Director, Rare Disease Institute | Children's National Hospital » speaking at Orphan Drug Congress

Deborah Requesens, Director, JumpStart, Orphan Disease Center, University of Pennsylvania, Perelman School of Medicine

Joshua Resnikoff, Chief Executive Officer, TMA precision health

Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries

Frank Rivera, President, Stronger Than Sarcoidosis

Brigitte Roberts, Director, Program Management, Comcast

Brigitte Roberts | Director, Program Management | Comcast » speaking at Orphan Drug Congress

Christopher Robertson, Professor of Law, Boston University

Derek Robertson, President, Maryland Sickle Cell Disease Association

Derek Robertson | President | Maryland Sickle Cell Disease Association » speaking at Orphan Drug Congress

Devin Rosenthal, Vice President & Head of Due Diligence, NovaQuest Capital Management

Heidi Ross, Vice President of Policy and Regulatory Affairs, National Organization for Rare Disorders

Ruth Rostron, Director of Program Management, Uniphar

Ruth Rostron | Director of Program Management | Uniphar » speaking at Orphan Drug Congress

Yury Rozenman, SVP of Business Development Life Sciences, Ada Health

Christopher Rudolf, Founder and Chief Executive Officer, volv global

Pierantonio Russo, FCCP, FAAP, STS, Corporate Chief Medical Officer, EVERSANA

Naomi Cohen Sacks, Vice President, HEORStrategies, A Division of ToxStrategies LLC

Naomi Cohen Sacks | Vice President | HEORStrategies, A Division of ToxStrategies LLC » speaking at Orphan Drug Congress

Naomi Sacks, Principal Scientist, EpidStrategies

Naomi Sacks | Principal Scientist | EpidStrategies » speaking at Orphan Drug Congress

Michael Salgaller, Supervisor, Technology Analysis and Marketing Unit (TAMU), National Institute of Health (NIH)

Matt Salo, Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer, Salo Health Strategies

Matt Salo | Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer | Salo Health Strategies » 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

Dick Salvatierra, President, Americas Health Foundation

Rachael Sawaya, Associate Director, Project Management – Execution, PCM Trials

Anthony Scatamacchia, Senior Vice President, Innovation, Patient Services, Eversana

Anthony Scatamacchia | Senior Vice President, Innovation, Patient Services | Eversana » speaking at Orphan Drug Congress

Mary Schaheen, President, Prevail Partners Llc

Charles Schmidt, Senior Medical Advisor, iNSTITUTO JÔ CLEMENTE

Matthias P. Schönermark, M.D., Ph., Managing Director, SKC Beratungsgesellschaft mbH

Carolyn Schwartz, President and Chief Scientist, DeltaQuest Foundation

Denise Scots Knight, Chief Executive Officer, Mereo Biopharma

Simone Seiter, Senior Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH

Albert Seymour, President & Chief Executive Officer, Homology Medicines

Albert Seymour | President & Chief Executive Officer | Homology Medicines » speaking at Orphan Drug Congress

Amit Sharma, Vice President Clinical Development - Global Therapeutic Area Head Nephrology & Hematology, Alexion Pharmaceuticals

Ryan Sheedy, Founder, mejo

Darlene Shelton, President/Founder, Danny’s Dose Alliance

Michael Sherman, MD, MBA, MS, Venture Partner, RA Capital Management

Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research

John Sholar, Chief Executive Officer, jCyte Inc

John Sholar | Chief Executive Officer | jCyte Inc » speaking at Orphan Drug Congress

Mridula Shukla, Head of Regulatory Affairs, Gene Therapy, Poseida Therapeutics, Inc.

Mridula Shukla | Head of Regulatory Affairs, Gene Therapy | Poseida Therapeutics, Inc. » speaking at Orphan Drug Congress

Amanda Singleton, Biopharma Partnerships, GeneDx

Jayson Slotnik, Partner, Health Policy Strategies, LLC

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

Rachel Smith, Global Head, Rare Disease, Parexel

Laura Smith van Carroll, Head of Insight & Advocacy, Metabolic Support UK

Oskar Smrzka, Chief Executive Officer/CSO, Ablevia

Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics

Rodica Stan, Senior Project Manager, NIH/NCATS

Paul Stanton, Senior Director, Global Strategy, Early Access, Inceptua Group

Paul Stanton | Senior Director, Global Strategy, Early Access | Inceptua Group » speaking at Orphan Drug Congress

Kim Stephens, Executive Director, Muenzer MPS Research & Treatment Center

Kim Stephens | Executive Director | Muenzer MPS Research & Treatment Center » speaking at Orphan Drug Congress

Dean Suhr, President and co-Founder, MLD Foundation

Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases

Jamie Sullivan | Vice President of Public Policy | EveryLife Foundation for Rare Diseases » speaking at Orphan Drug Congress

Donna Sullivan, Director Of Patient Advocacy, Pathways To Trust

Marshall Summar, Chief Executive Officer, Uncommon Cures

Marshall Summar | Chief Executive Officer | Uncommon Cures » speaking at Orphan Drug Congress

Laura Tadvalkar, Managing Director, RA Capital Management

Julia Taravella, Exececutive Director, Rare Trait Hope Fund

Julia Taravella | Exececutive Director | Rare Trait Hope Fund » speaking at Orphan Drug Congress

Jason Tardio, Chief Operating Officer, Ovid Therapeutics

Matt Teuteberg, President/ CEO, Splash Clinical

Pete Thornton, VP, Pharma Services & Strategic Partnerships, Invitae

Pete Thornton | VP, Pharma Services & Strategic Partnerships | Invitae » speaking at Orphan Drug Congress

Maria Törnsén, President, North America, Calliditas Therapeutics

Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center

Mark Trusheim | Strategic Director NEWDIGS, Center for Biomedical System Design | Tufts Medical Center » speaking at Orphan Drug Congress

Anthy Tsatoumas, Manager, Project Management, Veristat

Anthy Tsatoumas | Manager, Project Management | Veristat » 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

Nicholas Valle, Senior Product Director, iTakeControl, Red Nucleus

Nicholas Valle | Senior Product Director, iTakeControl | Red Nucleus » speaking at Orphan Drug Congress

Jytte van Huijstee, Director Clinical Trial Operations, myTomorrows

Jytte van Huijstee | Director Clinical Trial Operations | myTomorrows » speaking at Orphan Drug Congress

Leon van Wouwe, Clinical Innovation Director, volv global

Leon van Wouwe | Clinical Innovation Director | volv global » speaking at Orphan Drug Congress

Lavni Varyani, Founder, Pharma Business Partners

Lavni Varyani | Founder | Pharma Business Partners » speaking at Orphan Drug Congress

Geeta Vemuri, Managing Partner, Agent Capital

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

Louise Vetter, President & CEO, Huntington's Disease Society of America

Louise Vetter | President & CEO | Huntington's Disease Society of America » speaking at Orphan Drug Congress

Raghu Vishwanath, Global Head, Franchise Strategy and Operations, Rare Diseases Specialty Care, Sanofi

Raghu Vishwanath | Global Head,  Franchise Strategy and Operations, Rare Diseases Specialty Care | Sanofi » speaking at Orphan Drug Congress

Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio

Albert Wang, Co-founder, CEO, Comend

Albert Wang | Co-founder, CEO | Comend » speaking at Orphan Drug Congress

Mary Wang, Programme Director, Rare Diseases International

Mary Wang | Programme Director | Rare Diseases International » speaking at Orphan Drug Congress

Jon Weber, Senior Director of Cell & Gene Therapies Access Lead, Bayer

Michael Weickert, Chief Executive Officer, Pacylex Pharmaceuticals

Patricia Weltin, Chief Executive Officer, Beyond the Diagnosis

Melissa Williams, Founder, Simply Patient

Ben Willis, Director of Business Excellence, Egetis Therapeutics

Ben Willis | Director of Business Excellence | Egetis Therapeutics » speaking at Orphan Drug Congress

Richard Wilson, SVP North America Rare Disease Franchise Head, Kyowa Kirin

Kasey Woleben, Founder, Cure Mito Foundation/Rare Village

Kasey Woleben | Founder | Cure Mito Foundation/Rare Village » speaking at Orphan Drug Congress

Kristin Viswanathan Wolff, Senior Director, Global Government Affairs And Public Policy, bluebird bio

Kristin Viswanathan Wolff | Senior Director, Global Government Affairs And Public Policy | bluebird bio » speaking at Orphan Drug Congress

Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International

Durhane Wong-Rieger | President, Chief Executive Officer & Chair | Canadian Organisation for Rare Disorder & Rare Diseases International » speaking at Orphan Drug Congress

Justin Yang, Co-Founder, Kaibab Health & DMV Bio

Christine Yu, Chief Executive Officer, P2P Syncro

Samantha Zappia, MS, RAC, Principal Consultant, SK Regulatory Solutions

Samantha Zappia, MS, RAC | Principal Consultant | SK Regulatory Solutions » speaking at Orphan Drug Congress

Sophia Zilber, Board member, patient registry director, Cure Mito Foundation

Sophia Zilber | Board member, patient registry director | Cure Mito Foundation » speaking at Orphan Drug Congress

Get Involved At World Orphan Drug Congress USA

 

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