2025 Speakers

 

Mackenzie Abramson, Patient Advocate & Sr. Manager of Research Program Communications, Global Genes

Anthony Adamkovic, Executive Director, Pharmacovigilance, Harmony Biosciences, LLC

Anthony Adamkovic | Executive Director, Pharmacovigilance | Harmony Biosciences, LLC » speaking at Orphan Drug Congress

Dean Aguiar, Director Preclinical Research, TSC Alliance

Dean Aguiar | Director Preclinical Research | TSC Alliance » speaking at Orphan Drug Congress

Lynn Albizo, Chief Public Policy Officer, Immune Deficiency Foundation

Kathryn Alexander, Consultant, SMS Mom & Advocate

Amanda Almany, Parent Advisor, NKH Crusaders

Amanda Almany | Parent Advisor | NKH Crusaders » speaking at Orphan Drug Congress

Ahmad Alrawi, SVP Corporate Strategy, Commense Bio

Ahmad Alrawi | SVP Corporate Strategy | Commense Bio » speaking at Orphan Drug Congress

Kristin Archibald, Founder, NKH Crusaders

Ethan Ash, Senior VP of Business Development, BioNews

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

Leone Atkinson, Executive Medical Director, Rare Disease, Advanced Therapies and Pediatrics Team, Fortrea

Leone Atkinson | Executive Medical Director, Rare Disease, Advanced Therapies and Pediatrics Team | Fortrea » speaking at Orphan Drug Congress

Annette Bakker, CEO, Childrens Tumor Foundation

Ella Balasa, Patient Engagement Consultant, Patient Advocate

Matt Baniak, Vice President of Client and Delivery Excellence, EVERSANA

Tanya Bardakjian, Director, Medical Affairs Diagnostics, Sarepta Therapeutics

Tanya Bardakjian | Director, Medical Affairs Diagnostics | Sarepta Therapeutics » speaking at Orphan Drug Congress

Oliver Barnes, US Pharmaceutical & Biotech Correspondent, Financial Times

Oliver Barnes | US Pharmaceutical & Biotech Correspondent | Financial Times » speaking at Orphan Drug Congress

Erica Barnes, Rare Disease Advisory Council Administrator, University of Minnesota

Erica Barnes | Rare Disease Advisory Council Administrator | University of Minnesota » speaking at Orphan Drug Congress

Caitlyn Barrett, Director, Rare Disease and Oncology, Milken Institute Strategic Philanthropy Accelerator for Research and Collaboration (SPARC)

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

Thomas Bartlett, Rare Disease Patient Digital Technology Advisor, Bartlett Rare Disease Advocacy

Thomas Bartlett | Rare Disease Patient Digital Technology Advisor | Bartlett Rare Disease Advocacy » speaking at Orphan Drug Congress

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

Kevin Baruzzi | Partner | Simon-Kucher & Partners Strategy & Marketing Consultants GmbH » speaking at Orphan Drug Congress

Alison Bateman-House, Assistant Professor, Division of Medical Ethics, NYU Grossman School of Medicine

Alison Bateman-House | Assistant Professor, Division of Medical Ethics | NYU Grossman School of Medicine » speaking at Orphan Drug Congress

Andree Bates, Chief Executive Officer, Eularis Ltd

Andree Bates | Chief Executive Officer | Eularis Ltd » speaking at Orphan Drug Congress

Scott Baver, VP, Head of US Medical Affairs, ITF Therapeutics

Scott Baver | VP, Head of US Medical Affairs | ITF Therapeutics » speaking at Orphan Drug Congress

Anne Bechet, Global Head of Clinical Development and General Manager, Europe & USA, JCR Pharma

Natalie M. Beck, MGC, CGC, Senior Manager, Clinical Trials and RWD, Genome Medical

Natalie M. Beck | MGC, CGC, Senior Manager, Clinical Trials and RWD | Genome Medical » speaking at Orphan Drug Congress

Anny Bedard, President, Egetis Therapeutics US

Anny Bedard | President | Egetis Therapeutics US » speaking at Orphan Drug Congress

Hourinaz Behesti, Founder & CEO, Hebbian Bio

Hourinaz Behesti | Founder & CEO | Hebbian Bio » speaking at Orphan Drug Congress

Allyson Berent, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics, Foundation for Angelman Syndrome Therapeutics

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

Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research

Julieta B Bonvin Sallago, Lead Clinical Research Associate/ Assistant Professor, Connecticut Children's/ University of Connecticut School of medicine

Rohit Borker, Senior Vice President, Value Access, Health Economics & Outcomes Research, RTI Health Solutions

Rohit Borker | Senior Vice President, Value Access, Health Economics & Outcomes Research | RTI Health Solutions » speaking at Orphan Drug Congress

Lesa Brackbill, Associate Director of Advocacy, Patient Advocacy Strategies

Teresa Brandt, VP, Regulatory Affairs, Avidity Biosciences

Christine Brennan, Managing Director, Vertex Ventures HC

Christine Brennan | Managing Director | Vertex Ventures HC » speaking at Orphan Drug Congress

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

P.J. Brooks | Deputy Director of the NCATS Division of Rare Diseases Research Innovation | National Institutes of Health » speaking at Orphan Drug Congress

Benjamin Brown, Executive Director, American Society of Pharmacovigilance

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

Jonathan Bruce, Vice President, Market Access, Distribution, HEOR, F2G, Inc.

Mariana Burin, Medical Director, Thermo Fisher Scientific

Mariana Burin | Medical Director | Thermo Fisher Scientific » speaking at Orphan Drug Congress

Raquel Cabo, Head of Patient Advocacy, Communications, Commercial strategy, Ovid Therapeutics

Raquel Cabo | Head of Patient Advocacy, Communications, Commercial strategy | Ovid Therapeutics » speaking at Orphan Drug Congress

Melody Calkins, Director, Healthcare Policy, Biotechnology Innovation Organization

Mindy Cameron, President, AdvocacyWorks Consulting

Mindy Cameron | President | AdvocacyWorks Consulting » speaking at Orphan Drug Congress

Tanisha Carino, Visiting Fellow, Duke-Margolis Institute for Health Policy

Tanisha Carino | Visiting Fellow | Duke-Margolis Institute for Health Policy » speaking at Orphan Drug Congress

Janet Carlson, CEO, Creative Director, Copy, 1.11 Group

Joel Cartner, Director, Access Policy, Muscular Dystrophy Association

Cynthia Cassandro, Vice President, Patient Advoacy, Vigil neuroscience

Cynthia Cassandro | Vice President, Patient Advoacy | Vigil neuroscience » speaking at Orphan Drug Congress

Jill Castle, Director of Patient Education & Patient Advocacy, Little Hercules Foundation

Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics

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

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

James Chambers, Associate Professor, Tufts Medical Center

Fleur Chandler, Patient Advocate and Health Economist, Duchenne U.K.

Fleur Chandler | Patient Advocate and Health Economist | Duchenne U.K. » speaking at Orphan Drug Congress

Hayley Chapman, Senior Program Director, Patient Focused Drug Development

Timothy Chen, Medical Director, Caliway Biopharmaceuticals

Ellen Cho, Senior Director, Specialty Programs, Direct Relief

Ellen Cho | Senior Director, Specialty Programs | Direct Relief » speaking at Orphan Drug Congress

Duane Clark, General Manager, US Rare Disease, Sanofi

Duane Clark | General Manager, US Rare Disease | Sanofi » speaking at Orphan Drug Congress

Jim Coccia, Partner, TKG Access

Jason Colquitt, Chief Executive Officer, Across Healthcare

Amy Comstock Rick, Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA

Amy Comstock Rick | Director of Strategic Coalitions, Rare Disease Innovation Hub | FDA » speaking at Orphan Drug Congress

Rena Conti, Associate Professor, Markets, Public Policy, and Law, Boston University

Rena Conti | Associate Professor, Markets, Public Policy, and Law | Boston University » speaking at Orphan Drug Congress

Donna Cowan, Director EAP, Stealth BioTherapeutics

Donna Cowan | Director EAP | Stealth BioTherapeutics » speaking at Orphan Drug Congress

Glenna Crooks, PhD, Founder and CEO, Strategic Health Policy International, Inc

Andrew Cummins, VP Access and Reimbursement, Sciensus

Andrew Cummins | VP Access and Reimbursement | Sciensus » speaking at Orphan Drug Congress

Colleen Dansereau, RN, Vice President - Emerging Discoveries, Boston Children's Hospital

Marcella Debidda, President Patient Insights & Clinical Solutions, BioNews Clinical

Marcella Debidda | President Patient Insights & Clinical Solutions | BioNews Clinical » speaking at Orphan Drug Congress

Brigid DeCoursey Bondoc, Partner, Morrison Foerster

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

Marcus Delatte, VP, Regulatory Strategy, Allucent

Rumi Desai, Director, Global Regulatory Strategy, Amgen, Inc.

Rumi Desai | Director, Global Regulatory Strategy | Amgen, Inc. » speaking at Orphan Drug Congress

Veruska di Sena, SVP, Head of US Medical Affairs, Intercept Pharmaceuticals, Inc.

Veruska di Sena | SVP, Head of US Medical Affairs | Intercept Pharmaceuticals, Inc. » speaking at Orphan Drug Congress

Bradley Dickerson, VP of Project Management & Patient Advocacy, IECURE Inc

Elizabeth Donahue, Senior Director, Project Management, Thermo Fisher Scientific

Danielle Dong, Global Rare Medical Affairs, Scientific Advocacy Lead, Sanofi

Danielle Dong | Global Rare Medical Affairs, Scientific Advocacy Lead | Sanofi » speaking at Orphan Drug Congress

Sika Dunyoh, Senior Director of Patient Advocacy, Travere Therapeutics

Emma Eatwell, Global Practice Director, Health & Life Sciences, Global Counsel

Emma Eatwell | Global Practice Director, Health & Life Sciences | Global Counsel » speaking at Orphan Drug Congress

Heidi Edwards, President & Founder, Sisters’ Hope Foundation

Erika Edwards, Health and Medical Reporter and Writer, NBC News

Erika Edwards | Health and Medical Reporter and Writer | NBC News » speaking at Orphan Drug Congress

Michael Eging, Executive Director, Rare Access Action Project

Nevan Elam, Founder and Chief Executive Officer, Rezolute, Inc.

Nevan Elam | Founder and Chief Executive Officer | Rezolute, Inc. » speaking at Orphan Drug Congress

Jill Elliott, Vice President, Marketing, X4 Pharmaceuticals

Jill Elliott | Vice President, Marketing | X4 Pharmaceuticals » speaking at Orphan Drug Congress

Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics

Eric Feigen, Regulatory Affairs Manager, Immune Deficiency Foundation

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

Alicia Fiscus | Head of Global Regulatory Affairs and QA | Rhythm Pharmaceuticals » speaking at Orphan Drug Congress

Amber Fisher, Senior Clinical Research Manager, Kura Oncology Inc

Amber Fisher | Senior Clinical Research Manager | Kura Oncology Inc » speaking at Orphan Drug Congress

E'Lissa Flores, Director, Science & Regulatory Affairs, Biotechnology Innovation Organization

E'Lissa Flores | Director, Science & Regulatory Affairs | Biotechnology Innovation Organization » speaking at Orphan Drug Congress

Amanda Forys, Managing Partner, Magnolia Market Access

Pangkong Fox, Science Engagement Director, CACNA1A Foundation, Inc.

Pangkong Fox | Science Engagement Director | CACNA1A Foundation, Inc. » speaking at Orphan Drug Congress

Sheila Frame, President and Head of US Business, Gamida Cell Ltd

Karen Frascello, Senior Director, Medical Affairs, Alnylam Pharmaceuticals

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

Robert Fremeau, Founder and CEO, BrainStorm Therapeutics, Inc.

Stacey Frisk, Executive Director, Rare Disease Company Coalition

Stacey Frisk | Executive Director | Rare Disease Company Coalition » speaking at Orphan Drug Congress

Simon Frost, Chief Executive Officer & Co-Founder, Tiber Capital

Simon Frost | Chief Executive Officer & Co-Founder | Tiber Capital » speaking at Orphan Drug Congress

Suzanne Gaglianone, Patient Care Field Representative, ReveraGen BioPharma Inc

Suzanne Gaglianone | Patient Care Field Representative | ReveraGen BioPharma Inc » speaking at Orphan Drug Congress

Chris Garabedian, Portfolio Manager, Venture, Perceptive Advisors

Diego Garzon, VP, Corporate Development, Apertura Gene Therapy

Pamela Gavin, Chief Executive Officer, National Organization for Rare Disorders (NORD)

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

Victoria Gemme, Director of Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)

Victoria Gemme | Director of Policy and Regulatory Affairs | National Organization for Rare Disorders (NORD) » speaking at Orphan Drug Congress

Nayan Ghosh, Head of Life Sciences & HealthTech, Marwood Group

Nayan Ghosh | Head of Life Sciences & HealthTech | Marwood Group » speaking at Orphan Drug Congress

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

Anna Gilmore, Senior Director, Education & Advocacy, FSHD Society

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

Sonia Gobeil, Cofounder, Ataxia of Charlevoix-Saguenay Foundation

Josie Godfrey, Director, JG Zebra Consulting

Josie Godfrey | Director | JG Zebra Consulting » speaking at Orphan Drug Congress

Federico Goodsaid, SVP Regulatory and Quality, Ariana Pharma

J. Michael Graglia, Co-Founder & Managing Director, Syngap Research Fund

J. Michael Graglia | Co-Founder & Managing Director | Syngap Research Fund » speaking at Orphan Drug Congress

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

James Griffin, Sickle Cell Patient Advocate, Independent Milwaukee Wisconsin

Diego Guarin, Executive Director, Regional Market Access, Merck

Diego Guarin | Executive Director, Regional Market Access | Merck » speaking at Orphan Drug Congress

Kurt Gunter, Clinical Development Advisor, Steminent Biotherapeutics Inc.

Kurt Gunter | Clinical Development Advisor | Steminent Biotherapeutics Inc. » speaking at Orphan Drug Congress

David Gusick, Executive Director, Somebody To Talk To

David Gusick | Executive Director | Somebody To Talk To » speaking at Orphan Drug Congress

Kate Hanman, US Head of Rare Diseases, Costello Medical

Kate Hanman | US Head of Rare Diseases | Costello Medical » speaking at Orphan Drug Congress

Rachel Harrison, Director, Expanded Access Programs, Argenx

Rachel Harrison | Director, Expanded Access Programs | Argenx » speaking at Orphan Drug Congress

Christina Hartman, Senior Director, Government Affairs, Orchard therapeutics

Scott Harwood, Director, Check Rare

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

Atalah Haun, MD, Vice President, Medical Affairs, WEP Clinical

Atalah Haun, MD | Vice President, Medical Affairs | WEP Clinical » speaking at Orphan Drug Congress

Guadalupe Hayes Mota, CEO & Co-founder, Stealth Star-up & Senior Lecturer, Massachusetts Institute of Technology

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

Michele Herndon, Program Director, UDNF

Anne Heyes, Vice President, Head of Value Strategy, RTI Health Solutions

Karin Hoelzer, Senior Director, Patient Advocacy, Biotechnology Innovation Organization

Lauren Holder, Producer/Host, Help 4 HD Live Podcast, Help 4 HD

Lauren Holder | Producer/Host, Help 4 HD Live Podcast | Help 4 HD » speaking at Orphan Drug Congress

Elise Hoover, Vice President of Research, Foundation for Sarcoidosis Research

Patrick Hopkinson, Managing Director, 'International HTA Expert

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

Raymond Huml, Vice President, Rare & Orphan Strategy, Sciensus

Tim Hunt, Chief Executive Officer, Alliance for Regenerative Medicine

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

Mohammad Imran, Global BD Head, 3H Medi Solution Inc

Debra Jennings, Head of Patient Services Operations, North America, Kyowa Kirin

Gemma Jennings, Global Vice President of Programme Partnerships, BBC STORYWORKS

Zhou Jiang, VP of Early Commercial and Corporate Strategy, Edgewise Therapeutics

Zhou Jiang | VP of Early Commercial and Corporate Strategy | Edgewise Therapeutics » speaking at Orphan Drug Congress

Puja Joshi, Medical Director, Clinical Development, Stoke Therapeutics

Richie Kahn, Co-Founder & COO, Canary Advisors

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

Mwango Kashoki, MD, MPH, SVP, Global Head of Regulatory Strategy, Parexel

Joe Katakowski, Director of Research, RTW Foundation

Jessica Klein, Patient Advocacy & Industry Relations, Senior Liaison – East Region, Acadia Pharmaceuticals Inc.

Jessica Klein | Patient Advocacy & Industry Relations, Senior Liaison – East Region | Acadia Pharmaceuticals Inc. » speaking at Orphan Drug Congress

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

Jonathan Kornstein, VP, Rare Disease & Pediatrics Clinical Development, Caidya

Jonathan Kornstein | VP,  Rare Disease & Pediatrics Clinical Development | Caidya » speaking at Orphan Drug Congress

Graeme Ladds, CEO & Owner, PharSafer

Graeme Ladds | CEO & Owner | PharSafer » speaking at Orphan Drug Congress

Patroski Lawson, Co-founder, The Just Society

Patroski Lawson | Co-founder | The Just Society » speaking at Orphan Drug Congress

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

Kerry Lee, Assoc. Director for Rare Diseases, Food and Drug Administration

Kerry Lee | Assoc. Director for Rare Diseases | Food and Drug Administration » speaking at Orphan Drug Congress

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

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

Jason Lettiere, Senior Vice President, Corporate and Business Development, Neurvati Neurosciences

Freda C Lewis-Hall, Former Chief Patient Officer And Executive Vice President, Pfizer

Freda C Lewis-Hall | Former Chief Patient Officer And Executive Vice President | Pfizer » speaking at Orphan Drug Congress

Michael Lingzhi Li, Assistant Professor of Business Administration, Harvard Business School, Technology and Operations Management

Naomi Litchfield, Director, Patient Advocacy, Bionical Emas

Andrew Lo, Charles E. and Susan T. Harris Professor, MIT Sloan School of Management

Andrew Lo | Charles E. and Susan T. Harris Professor | MIT Sloan School of Management » speaking at Orphan Drug Congress

Mathieu Loiseau, Director of Rare Clinical Services, Sciensus

Mathieu Loiseau | Director of Rare Clinical Services | Sciensus » speaking at Orphan Drug Congress

Veronica Lopez Gousset, MPH, Steering Committee Member,, HTAi Rare Disease Interest Group

Peng Lu, Chief Medical Officer, Pharvaris

Alastair MacDonald, Vice President, Medical Affairs, Sciensus

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

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

Victor Maertens, Government Affairs Director, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

Victor Maertens | Government Affairs Director | European Confederation of Pharmaceutical Entrepreneurs (Belgium) » speaking at Orphan Drug Congress

Alexandra Mangili, Head of Clinical Sciences, Alfasigma

Alexandra Mangili | Head of Clinical Sciences | Alfasigma » speaking at Orphan Drug Congress

Yuri Maricich, MD, MBA;, Chief Medical Officer, CAMP4 Therapeutics

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

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

Craig Martin, Founder, CEO, Orphan Therapeutics Accelerator

Craig Martin | Founder, CEO | Orphan Therapeutics Accelerator » speaking at Orphan Drug Congress

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

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

Fabiana Marzabal, Sr Director Forecasting, Analytics and Insights, Viridian Therapeutics

Cara Mayfield, VP, Corporate Affairs, Neurogene

Maurizio Mazzi, Senior Associate Director, Patient Advocacy & Professional Relations, Böhringer Ingelheim

Reenie McCarthy, Chief Executive Officer, Stealth BioTherapeutics

Katie McCarthy, SVP & Practice Lead, Clinical & Regulatory, Lumanity

Alice McConnell, Chief Executive Officer, Speragen, Inc

Alice McConnell | Chief Executive Officer | Speragen, Inc » 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

Jennifer McNary, Co-Founder, Canary Advisors

Jennifer McNary | Co-Founder | Canary Advisors » speaking at Orphan Drug Congress

Laura McWright, Deputy Director, Seamless Care Models Group/ Centers for Medicare and Medicaid Services

Laura McWright | Deputy Director | Seamless Care Models Group/ Centers for Medicare and Medicaid Services » speaking at Orphan Drug Congress

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

Ana Mingorance, Chief Development Officer, Loulou Foundation

Maryam Mokhtarzadeh, Senior Director Regulatory Strategy, REGENXBIO

Juliet Moritz, Senior VP Strategic Solutions and Patient Centricity, Ergomed

Juliet Moritz | Senior VP Strategic Solutions and Patient Centricity | Ergomed » speaking at Orphan Drug Congress

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

Rachit Mukkamala, President, MIT Biotech Group, Massachusetts Institute of Technology

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

Ramaiah Muthyala | Professor/Director at University of Minnesota, President and Chief Executive Officer, | Indian organization for Rare diseases » speaking at Orphan Drug Congress

Brian Nappi, SVP, Strategy, Saol Therapeutics

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

Amy Nicole Nayar, Vice President of U.S. Patient Advocacy & Government Affairs, Novartis Gene Therapies

Kaylee Nguyen, Conference Producer, Terrapinn

Kaylee Nguyen | Conference Producer | Terrapinn » speaking at Orphan Drug Congress

Katie Niemeyer, Speaker, Philanthropist, CRNA, SJS Survivor, Katie Niemeyer, Speaker LLC

Megan Nolan, CEO, Editor-in-Chief, Rare Parenting

Megan Nolan | CEO, Editor-in-Chief | Rare Parenting » speaking at Orphan Drug Congress

Kevan Oconnor, Venture Investor, Cystic Fibrosis Foundation

Eunhee Ohgami, CEO, NeuroCores Inc,

Eunhee Ohgami | CEO | NeuroCores Inc, » speaking at Orphan Drug Congress

Paula Orandash, Director, Patient Engagement, Ergomed

Michele M Oshman, Chief Patient Advocate & Head, Patient Advocacy Center of Excellence, Biotechnology Innovation Organization

Francis Pang, SVP Global Market Access and International Geographic Expansion, Orchard therapeutics

Francis Pang | SVP Global Market Access and International Geographic Expansion | Orchard therapeutics » speaking at Orphan Drug Congress

Alexandre Pare, Scientific Researcher, McGill University

Alexandre Pare | Scientific Researcher | McGill University » speaking at Orphan Drug Congress

Effie Parks, Podcast Host, CTNNB1 Connect & Cure, Once Upon A Gene - CTNNB1

Effie Parks | Podcast Host,  CTNNB1 Connect & Cure | Once Upon A Gene - CTNNB1 » speaking at Orphan Drug Congress

Simrit Parmar, Founder, cellenkos

Kinnari Patel, President, Head of R&D and COO, Rocket Pharma

Doug Paul, VP Commercial Strategy, Cypress Biopharma

Doug Paul | VP Commercial Strategy | Cypress Biopharma » speaking at Orphan Drug Congress

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

Dave Pearce | Chair | International Rare Diseases Research Consortium (IRDiRC) » speaking at Orphan Drug Congress

Allison Peck, Co-Foundet, Cure VCP Disease

Melissa Penn, Director Patient Engagement Research & Development, Bayer

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

Mike Porath, Founder And Chief Executive Officer, The mighty

Mike Porath | Founder And Chief Executive Officer | The mighty » speaking at Orphan Drug Congress

Bryce Powerman, Director, Business Development, Natera

Bryce Powerman | Director, Business Development | Natera » speaking at Orphan Drug Congress

Matthew Prentice, Director of State Policy, Immune Deficiency Foundation

Matthew Prentice | Director of State Policy | Immune Deficiency Foundation » speaking at Orphan Drug Congress

Raymond Puerini, Director, FasterCures, a Center of the Milken Institute

Donovan Quill, EVP of Life Science Strategies, AscellaHealth

Maha Radhakrishnan, Executive Partner, Sofinnova Investments

Harsha Rajasimha, Founder and Chairman, IndoUSRare

Harsha Rajasimha | Founder and Chairman | IndoUSRare » speaking at Orphan Drug Congress

Patrick Raletz, Project Manager, EAP, Bionical Emas

Harpreet Ram, President, EVR Consulting

Dhaval Rathod, Associate Director, Pharm Sci, Shionogi Inc

Dorian Readnour, Vice President, International, Sarepta Therapeutics

Dorian Readnour | Vice President, International | Sarepta Therapeutics » speaking at Orphan Drug Congress

Lindsay Reel, VP, Pharmacy Practice & Specialty Solutions, EVERSANA

Rebecca Reimers, Medical Geneticist, Rady Children's Institute for Genomic Medicine

Deborah Requesens, Founder, Society of Hispanic Rare Diseases

Faisal Riaz, Medical Director Neuroimmunology and AATD, Takeda Pharmaceuticals USA Inc

Sumira Riaz, Health Psychologist & Patient Engagement Consultant, Unboxed Psychology

Sara Riordan, Vice President, Strategy, Genome Medical

Sara Riordan | Vice President, Strategy | Genome Medical » speaking at Orphan Drug Congress

Frank Rivera, President, Stronger Than Sarcoidosis

Frank Rivera | President | Stronger Than Sarcoidosis » speaking at Orphan Drug Congress

Luke Rosen, Founder, Rescue 7: Firefighters for Patients

Johanna Rossell, Senior Vice President, General Manager, Sumitomo pharma

Seth Rotberg, Sr. Manager, Patient Advocacy and Engagement, Prilenia

Christian Rubio, Executive Director, EverythingALS

Christopher Rudolf, Founder and Chief Executive Officer, Volv Global

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

Matthew Salo, President, Salo Health Strategies, Salo Health Strategies

Marina Sardone, Global Head of Patient Advocacy, Alfasigma

William Sarraille, Adjunct Professor, University of Maryland Francis King Carey School of Law

William Sarraille | Adjunct Professor | University of Maryland Francis King Carey School of Law » speaking at Orphan Drug Congress

Mary Schaheen, President, Prevail Partners Llc

Jeremy Schmahmann, Founding Director, Massachusetts General Hospital Ataxia Center, Massachusetts General Hospital Ataxia Center

Jeremy Schmahmann | Founding Director, Massachusetts General Hospital Ataxia Center | Massachusetts General Hospital Ataxia Center » speaking at Orphan Drug Congress

Jörg Schüttrumpf, Chief Scientific Innovation Officer, Grifols

Jörg Schüttrumpf | Chief Scientific Innovation Officer | Grifols » speaking at Orphan Drug Congress

Carolyn Schwartz, President & Chief Scientist, DeltaQuest Foundation

Denise Scots Knight, Chief Executive Officer, Mereo Biopharma

Jessica Scott, Chief Executive Officer, Legacy Health Strategies

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

Simone Seiter | Senior Partner | Simon-Kucher & Partners Strategy & Marketing Consultants GmbH » speaking at Orphan Drug Congress

Kathy Senecal, Rare Disease Patient

Kathy Senecal |  | Rare Disease Patient » speaking at Orphan Drug Congress

Seungoh Seo, Senior AI Researcher, ONCOCROSS

Seungoh Seo | Senior AI Researcher | ONCOCROSS » speaking at Orphan Drug Congress

Bridget Seritt, Founder, Advocates for Compassionate Therapy Now

Bridget Seritt | Founder | Advocates for Compassionate Therapy Now » speaking at Orphan Drug Congress

Andrew Shao, SVP of Global Scientific and Regulatory Affairs, Niagen Biosceince

Jordan Shin, Chief Medical Officer, HAYA Therapeutics

Jordan Shin | Chief Medical Officer | HAYA Therapeutics » speaking at Orphan Drug Congress

Tricha Shivas, Chief Staff and Strategy, Foundation for Sarcoidosis Research

John Sholar, Chief Executive Officer, jCyte Inc

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

Jennifer Shumsky, Founder and Principal, JLS Consulting LLC

Jennifer Shumsky | Founder and Principal | JLS Consulting LLC » speaking at Orphan Drug Congress

Amanda Singleton, Medical Affairs Director, Biopharma & Patient Advocacy, GeneDx

Amanda Singleton | Medical Affairs Director, Biopharma & Patient Advocacy | GeneDx » speaking at Orphan Drug Congress

Ted Slocomb, Vice President, Global Commercial, Value & Access (frmr.) Kite Pharma, Managing Partner, TS BioAdvisors

Jayson Slotnik, Partner, Health Policy Strategies, LLC

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

Kimberly Smith, Chief Medical Officer, FDA/CBER

Kimberly Smith | Chief Medical Officer | FDA/CBER » speaking at Orphan Drug Congress

Rachel Smith, Executive Director, Global Head, Rare Disease, Parexel

Jennifer Snow, Founder and Principal, Apteka LLC

Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics

Frank Sorgi | President and Chief Executive Officer | FLAG Therapeutics » speaking at Orphan Drug Congress

Mangala Soundar, Senior Director, Discovery Neurology,, PepGen

Mangala Soundar | Senior Director, Discovery Neurology, | PepGen » speaking at Orphan Drug Congress

Steve St. Onge, SVP - Corporate Development, Clarametyx Biosciences

Dionne Stalling, Executive Director, Rare And Black

Dionne Stalling | Executive Director | Rare And Black » speaking at Orphan Drug Congress

John Stanford, Managing Director, Prism Group

John Stanford | Managing Director | Prism Group » speaking at Orphan Drug Congress

Stephanie Steele, Director, Immune Deficiency Foundation

Andrew Steinsapir, Acting Chief Technology Officer, Apertura Gene Therapy

Andrew Steinsapir | Acting Chief Technology Officer | Apertura Gene Therapy » speaking at Orphan Drug Congress

Swami Subramanian, Sr. Director, US/Global Pricing, Regeneron Pharmaceuticals Inc

Swami Subramanian | Sr. Director, US/Global Pricing | Regeneron Pharmaceuticals Inc » speaking at Orphan Drug Congress

Hiroshi Sugimoto, Director, Takeda Pharmaceuticals

Donna Sullivan, Director of patient advocacy, Pathways to Trust

Erin Sullivan, Executive Director, Sisters’ Hope Foundation

Marshall Summar, Chief Executive Officer, Uncommon Cures

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

Jean Swidler, Executive Director, End the Legacy

Jean Swidler | Executive Director | End the Legacy » speaking at Orphan Drug Congress

Amy Teets, Global Head Patient Strategy, Specialty Care, Sanofi

Amy Teets | Global Head Patient Strategy, Specialty Care | Sanofi » speaking at Orphan Drug Congress

Pierre A Teulié, CEO, ODDIFACT

Pierre A Teulié | CEO | ODDIFACT » speaking at Orphan Drug Congress

Betsy Trainor, Board Member, Ataxia of Charlevoix-Saguenay Foundation

Chelsea Trengrove, CEO and Cofounder, Neoclease

Chelsea Trengrove | CEO and Cofounder | Neoclease » speaking at Orphan Drug Congress

Matt Trudeau, President, ITF Therapeutics

Karen Tubridy, Chief Operating Officer, Abcuro, Inc.

Leon van Wouwe, Clinical Innovation Director, Volv Global

Surabhi Verma, Chief Medical Officer, Leadiant Biosciences

Surabhi Verma | Chief Medical Officer | Leadiant Biosciences » speaking at Orphan Drug Congress

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

Daniel Wainstock | Researcher and Rare Disease Advocate | PUC-Rio » speaking at Orphan Drug Congress

Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition, BWHI

Jenifer Waldrop | Executive Director | Rare Disease Diversity Coalition, BWHI » speaking at Orphan Drug Congress

Colin Werth, Pharma Consultant, Patient Advocate

Melissa Williams, CEO, Simply Patient

Susan Winckler, Chief Executive Officer, Reagan Udall Foundation for the F.D.A.

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

Jodi Wolff, Founder, Strategic Advocacy Solutions, LLC

Jodi Wolff | Founder | Strategic Advocacy Solutions, LLC » speaking at Orphan Drug Congress

Rosangela Wolff Moro, Congresswoman, Federal Chamber of Deputies, São Paulo, Brazil

Rosangela Wolff Moro | Congresswoman | Federal Chamber of Deputies, São Paulo, Brazil » speaking at Orphan Drug Congress

Sorrel Wolowacz, Vice President, R.T.I. Health Solutions

Sorrel Wolowacz | Vice President | R.T.I. Health Solutions » speaking at Orphan Drug Congress

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

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

Susan Woolner, Hauenstein Neurosciences Patient & Caregiver Support/Community Partner Manager, Trinity Health MI

Susan Woolner | Hauenstein Neurosciences Patient & Caregiver Support/Community Partner Manager | Trinity Health MI » speaking at Orphan Drug Congress

Alyssa Wyant, Chief Regulatory & Quality Officer, Praxis Precision Medicines

Alyssa Wyant | Chief Regulatory & Quality Officer | Praxis Precision Medicines » speaking at Orphan Drug Congress

David Young, President of R&D, Processa Pharmaceuticals, Inc.

Joseph Zabinski, VP, Head of Commercial Strategy & AI, OM1

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

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

Sophia Zilber, Vice President, Cure Mito Foundation

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