2023 Speakers

 

Faruk Abdullah, President, Professional Services and Chief Business Officer, EVERSANA

Faruk Abdullah | President, Professional Services and Chief Business Officer | EVERSANA » speaking at Orphan Drug Congress

Suchismita Acharya, Chief Executive Officer, AyuVis Research

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

Rania Al Shami, Chief Business and Strategy Officer, PDC CRO

Rania Al Shami | Chief Business and Strategy Officer | PDC CRO » speaking at Orphan Drug Congress

Meg Alexander, Corporate Affairs Officer, Ovid Therapeutics, Ovid Therapeutics

Meg Alexander | Corporate Affairs Officer, Ovid Therapeutics | Ovid 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

Irina Antonijevic, Chief Medical Officer, EveryONE Medicines

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

Andrew Auruku, Chief Executive Officer, Rare Diseases Uganda-RDU

Andrew Auruku | Chief Executive Officer | Rare Diseases Uganda-RDU » speaking at Orphan Drug Congress

Rena Baek, Senior Director - Rare Diseases, Moderna, Inc.

Rena Baek | Senior Director - Rare Diseases | Moderna, Inc. » speaking at Orphan Drug Congress

Ritu Baral, Managing Director And Senior Biotechnology Analyst, Cowen

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

Kelly Barta, President, Coalition of Skin Diseases

Kelly Barta | President | Coalition of Skin Diseases » speaking at Orphan Drug Congress

Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance

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

Norman Barton, Chair of the Clinical and Scientific Advisory Board, Oak Hill Bio

Norman Barton | Chair of the Clinical and Scientific Advisory Board | Oak Hill Bio » speaking at Orphan Drug Congress

Kevin Baruzzi, Partner, Simon-Kucher

Kevin Baruzzi | Partner | Simon-Kucher » speaking at Orphan Drug Congress

Mark Battaglini, Chief Strategy Officer, Alliance for Regenerative Medicine

Mark Battaglini | Chief Strategy Officer | Alliance for Regenerative Medicine » 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

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

Dawn Berry, President and Co-Founder, LunaPBC

Dawn Berry | President and Co-Founder | LunaPBC » speaking at Orphan Drug Congress

Sulagna Bhattacharya, Chief Executive Officer, NanoScope Technologies

Sulagna Bhattacharya | Chief Executive Officer | NanoScope Technologies » speaking at Orphan Drug Congress

Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.

Kathie Bishop | SVP, Head of Rare Disease and External Innovation | Acadia Pharmaceuticals Inc. » speaking at Orphan Drug Congress

Henric Bjarke, Chief Operating Officer, Inozyme

Henric Bjarke | Chief Operating Officer | Inozyme » speaking at Orphan Drug Congress

Fabrice Bocquillon, Global Head, Digital Specialty Care, Sanofi

Fabrice Bocquillon | Global Head, Digital Specialty Care | Sanofi » 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

Wendy Borsari, Senior Manager of Patient Advocacy, Tenaya Therapeutics

Wendy Borsari | Senior Manager of Patient Advocacy | Tenaya Therapeutics » speaking at Orphan Drug Congress

Pierre-Olivier Boyer, Country Manager, PTC Therapeutics

Pierre-Olivier Boyer | Country Manager | PTC Therapeutics » speaking at Orphan Drug Congress

Marie Brace, Patient Access Liaison, Catalyst Pharmaceutical Partners

Marie Brace | Patient Access Liaison | Catalyst Pharmaceutical Partners » speaking at Orphan Drug Congress

Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

Julie Breneiser | Executive Director | Gorlin Syndrome Alliance » speaking at Orphan Drug Congress

Matthias Breugelmans, Chief Executive Officer, Elastrin Therapeutics

Matthias Breugelmans | Chief Executive Officer | Elastrin Therapeutics » speaking at Orphan Drug Congress

Rusty Bromley, Director of Operations, AyuVis Research, Inc.

Rusty Bromley | Director of Operations | AyuVis Research, Inc. » speaking at Orphan Drug Congress

P.J. Brooks, Acting Director, Division of Rare Diseases Research Innovation, NIH/NCATS

P.J. Brooks | Acting Director, Division of Rare Diseases Research Innovation | NIH/NCATS » speaking at Orphan Drug Congress

Robert Buchanan, Founder And Partner, Ventac Partners

Robert Buchanan | Founder And Partner | Ventac Partners » speaking at Orphan Drug Congress

Raquel Cabo, VP, Global Head of Pricing & Market Access, Ovid Therapeutics

Raquel Cabo | VP, Global Head of Pricing & Market Access | Ovid Therapeutics » 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

Leonardo Calzetti, Co-founding Director & Sr. Partner, BlueDil International

Leonardo Calzetti | Co-founding Director & Sr. Partner | BlueDil International » speaking at Orphan Drug Congress

Tanisha Carino, Board Member, Every Cure

Tanisha Carino | Board Member | Every Cure » speaking at Orphan Drug Congress

Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics

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

John Cavitt, Parent, BCM Families Foundation

John Cavitt | Parent | BCM Families Foundation » speaking at Orphan Drug Congress

Afshawn Chakamian, Director of Therapeutic Expertise, ICON

Afshawn Chakamian | Director of Therapeutic Expertise | ICON » speaking at Orphan Drug Congress

Anne-Sophie Chalandon, Head of Rare Diseases Global Public Affairs Rare Disease and ATMP Policy at Sanofi, Vice-Chair of the EFPIA OMP WG, Sanofi

Anne-Sophie Chalandon | Head of Rare Diseases Global Public Affairs Rare Disease and ATMP Policy at Sanofi, Vice-Chair of the EFPIA OMP WG | Sanofi » speaking at Orphan Drug Congress

David Chapman, Vice President of Market Access, Agios Pharmaceuticals

David Chapman | Vice President of Market Access | Agios Pharmaceuticals » speaking at Orphan Drug Congress

Richard Chapman, Chief Science Officer, Innovation and Value Initiative

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

Jonathan CHAPPER, CHAPPER healthcare - CEO, EUROPACCESS

Jonathan CHAPPER | CHAPPER healthcare - CEO | EUROPACCESS » speaking at Orphan Drug Congress

Yi-Chun Maria Chen, VP of Business Development, BRIM Biotechnology

Yi-Chun Maria Chen | VP of Business Development | BRIM Biotechnology » speaking at Orphan Drug Congress

Nimi Chhina, Head, Global R&D and Regulatory Policy, BioMarin Pharmaceutical Inc.

Nimi Chhina | Head, Global R&D and Regulatory Policy | BioMarin Pharmaceutical Inc. » speaking at Orphan Drug Congress

Nitin Choudhary, Vice President, IPM.ai

Nitin Choudhary | Vice President | IPM.ai » speaking at Orphan Drug Congress

Steven Cohen, Executive Director, Translational Medicine-Oncology & Internal Medicine, & Search & Evaluation-IM, Daiichi Sankyo

Steven Cohen | Executive Director, Translational Medicine-Oncology & Internal Medicine, & Search & Evaluation-IM | Daiichi Sankyo » speaking at Orphan Drug Congress

Cathy Collet, ALS Caregiver, Patient Advocate

Cathy Collet | ALS Caregiver | Patient Advocate » speaking at Orphan Drug Congress

Rachelle Cook, Community Manager, TREND Community

Rachelle Cook | Community Manager | TREND Community » speaking at Orphan Drug Congress

Ashley Cox, Senior Principal, Head of Europe | President HBA - London Chapter, EVERSANA

Ashley Cox | Senior Principal, Head of Europe | President HBA - London Chapter | EVERSANA » speaking at Orphan Drug Congress

Laura Cox, Director Partnerships, myTomorrows

Laura Cox | Director Partnerships | myTomorrows » speaking at Orphan Drug Congress

Carole Cramer, Chief Executive Officer and CoFounder, Biostrategies

Carole Cramer | Chief Executive Officer and CoFounder | Biostrategies » speaking at Orphan Drug Congress

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

Charlotte Damiano, Global Regulatory Partner, Life Sciences, Hogan Lovells

Charlotte Damiano | Global Regulatory Partner, Life Sciences | Hogan Lovells » speaking at Orphan Drug Congress

Mark Dant, Executive Director, The Ryan Foundation

Mark Dant | Executive Director | The Ryan Foundation » speaking at Orphan Drug Congress

Kristina Davidson, Chief Corporate Affairs Officer, Horizon Therapeutics

Kristina Davidson | Chief Corporate Affairs Officer | Horizon Therapeutics » speaking at Orphan Drug Congress

Kendall Davis, Director, Advocacy & Engagement Strategy Center for Rare Diseases, ICON

Kendall Davis | Director, Advocacy & Engagement Strategy Center for Rare Diseases | ICON » speaking at Orphan Drug Congress

Michelle Davis, Executive Director, International Fibrodysplasia Ossificans Progressiva Association

Michelle Davis | Executive Director | International Fibrodysplasia Ossificans Progressiva Association » speaking at Orphan Drug Congress

Sugato De, VP, Regulatory Strategy, Head of MedTech, Allucent

Sugato De | VP, Regulatory Strategy, Head of MedTech | Allucent » speaking at Orphan Drug Congress

Laurence Dean, Managing Member, Twin Spires Consulting LLC

Laurence Dean | Managing Member | Twin Spires Consulting LLC » 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

Christopher DeFelice, Founder and CTO, TREND Community

Christopher DeFelice | Founder and CTO | TREND Community » speaking at Orphan Drug Congress

Maria Della Rocca, Sr. Director, Patient Support & Education, Global Genes

Maria Della Rocca | Sr. Director, Patient Support & Education | Global Genes » speaking at Orphan Drug Congress

Lisa Deschamps, Chief Executive Officer, AviadoBio

Lisa Deschamps | Chief Executive Officer | AviadoBio » speaking at Orphan Drug Congress

Patricia Dickson, Centennial Professor of Pediatrics, Professor of Genetics, Chief, Division of Genetics and Genomic Medicine, Washington University

Patricia Dickson | Centennial Professor of Pediatrics, Professor of Genetics, Chief, Division of Genetics and Genomic Medicine | Washington University » speaking at Orphan Drug Congress

Deb Discenza, Chief Executive Officer, PreemieWorld

Deb Discenza | Chief Executive Officer | PreemieWorld » speaking at Orphan Drug Congress

Victoria Dohnal, Associate Director, Global Regulatory Policy & Intelligence, Janssen

Victoria Dohnal | Associate Director, Global Regulatory Policy & Intelligence | Janssen » 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

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

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

Michael Dudley, Chief Executive Officer and Co-founder, TransCode Therapeutics

Michael Dudley | Chief Executive Officer and Co-founder | TransCode Therapeutics » 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

Patrick Dunn, Executive Director, International Pemphigus Pemphigoid Foundation

Patrick Dunn | Executive Director | International Pemphigus Pemphigoid Foundation » speaking at Orphan Drug Congress

Abie Ekangaki, Vice President, Statistical Consulting, Premier Research

Abie Ekangaki | Vice President, Statistical Consulting | Premier Research » speaking at Orphan Drug Congress

Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals

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

Shaun Ellis, Vice President, Global Market Access CVM Retina, Janssen (J&J)

Shaun Ellis | Vice President, Global Market Access CVM Retina | Janssen (J&J) » speaking at Orphan Drug Congress

Ben Enejo, Partner - Life Sciences, Arthur D. Little

Ben Enejo | Partner - Life Sciences | Arthur D. Little » 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

Lisa Feng, Senior Director, Health Policy, Alexion – AstraZeneca Rare Disease

Lisa Feng | Senior Director, Health Policy | Alexion – AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Holly Fernandez Lynch, Department of Medical Ethics & Health Policy, University of Pennsylvania

Holly Fernandez Lynch | Department of Medical Ethics & Health Policy | University of Pennsylvania » speaking at Orphan Drug Congress

Nasha Fitter, VP Patient Network & Data, and Co-Founder & CEO, FOXG1 Research Foundation, Invitae

Nasha Fitter | VP Patient Network & Data, and Co-Founder & CEO, FOXG1 Research Foundation | Invitae » speaking at Orphan Drug Congress

Julie Fleshman, President And Chief Executive Officer, Pancreatic Cancer Action Network

Julie Fleshman | President And Chief Executive Officer | Pancreatic Cancer Action Network » speaking at Orphan Drug Congress

Mark Fleury, Principal, Policy Development - Emerging Science, American Cancer Society Action Network

Mark Fleury | Principal, Policy Development - Emerging Science | American Cancer Society Action Network » speaking at Orphan Drug Congress

Max Flurie, Senior Data Scientist, Trend

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

Sylvain Forget, Chief Executive Officer, BlueDil International

Sylvain Forget | Chief Executive Officer | BlueDil International » speaking at Orphan Drug Congress

Miranda Franco, Senior Policy Advisor, Holland & Knight

Miranda Franco | Senior Policy Advisor | Holland & Knight » speaking at Orphan Drug Congress

Albert Freedman, Rare Father/Counseling Psychologist, rarecounseling.com

Albert Freedman | Rare Father/Counseling Psychologist | rarecounseling.com » speaking at Orphan Drug Congress

Neha Gandhi, Head of Global Patient Advocacy and Engagment, Ascendis Pharma

Neha Gandhi | Head of Global Patient Advocacy and Engagment | Ascendis Pharma » 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

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

Laura Genoves, TREND Community

Laura Genoves |  | TREND Community » speaking at Orphan Drug Congress

Michele Gerber, Chief Medical Officer, Myeloid Therapeutics

Michele Gerber | Chief Medical Officer | Myeloid Therapeutics » 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

Robert Goldstein, Associate Director, Global Patient Advocacy, Neuromuscular, Biogen

Robert Goldstein | Associate Director, Global Patient Advocacy, Neuromuscular | Biogen » speaking at Orphan Drug Congress

Mari Gonzalez, Community Member, TREND Community

Mari Gonzalez | Community Member | TREND Community » speaking at Orphan Drug Congress

Mike Graglia, Founder & CEO, Syngap Research Fund

Mike Graglia | Founder & CEO | Syngap Research Fund » speaking at Orphan Drug Congress

Alicia Granados, Global Head of Rare Disease Medical Scientific Advocacy & Insights, Sanofi

Alicia Granados | Global Head of Rare Disease Medical Scientific Advocacy & Insights | Sanofi » speaking at Orphan Drug Congress

Frederieke Grevers, Medical Operations Manager, myTomorrows

Frederieke Grevers | Medical Operations Manager | myTomorrows » speaking at Orphan Drug Congress

Erin Griner, Assistant Director of Clinical Research Methodology, Worldwide Clinical Trials

Erin Griner | Assistant Director of Clinical Research Methodology | Worldwide Clinical Trials » speaking at Orphan Drug Congress

Amy Grover, Director of Patient Advocacy, Catalyst Pharmaceutical Partners

Amy Grover | Director of Patient Advocacy | Catalyst Pharmaceutical Partners » speaking at Orphan Drug Congress

Maria Hadjidemetriou, Specialist Management Account, Medscheme Holdings (Pty) Ltd

Maria Hadjidemetriou | Specialist Management Account | Medscheme Holdings (Pty) Ltd » speaking at Orphan Drug Congress

Alix Hall, Patient Advocacy Manager, Bionical Emas

Alix Hall | Patient Advocacy Manager | Bionical Emas » speaking at Orphan Drug Congress

Alaa Hamed, Global Head of Medical Affairs Rare Diseases, Sanofi

Alaa Hamed | Global Head of Medical Affairs Rare Diseases | Sanofi » speaking at Orphan Drug Congress

Nicolas HANSL, ILAPO - Key Account Manager Clinical Trials and EAP, EUROPACCESS

Nicolas HANSL | ILAPO - Key Account Manager Clinical Trials and EAP | EUROPACCESS » speaking at Orphan Drug Congress

Bree Harlin, Chief Clinical Officer, TC Biopharm

Bree Harlin | Chief Clinical Officer | TC Biopharm » speaking at Orphan Drug Congress

Molly Harper, Chief Business Officer, Synlogic

Molly Harper | Chief Business Officer | Synlogic » speaking at Orphan Drug Congress

Rachel Harrison, Associate Director, Early Access Programs, Apellis Pharmaceuticals, Inc

Rachel Harrison | Associate Director, Early Access Programs | Apellis Pharmaceuticals, Inc » speaking at Orphan Drug Congress

Scott Harwood, Director, Check Rare

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

Lauren Heiser, Business Development Director, Clinigen Group

Lauren Heiser | Business Development Director | Clinigen Group » speaking at Orphan Drug Congress

Terry Hermiston, Chief Executive Officer, Coagulant Therapeutics

Terry Hermiston | Chief Executive Officer | Coagulant Therapeutics » speaking at Orphan Drug Congress

Sharon Hesterlee, Chief Research Officer, MDA

Sharon Hesterlee | Chief Research Officer | MDA » speaking at Orphan Drug Congress

Kenneth Hobby, President, Cure SMA

Kenneth Hobby | President | Cure SMA » speaking at Orphan Drug Congress

Karin Hoelzer, Director of Policy and Regulatory Affairs, National Organization for Rare Disorders

Karin Hoelzer | Director of Policy and Regulatory Affairs | National Organization for Rare Disorders » speaking at Orphan Drug Congress

Lori Irvin, NICU RN and Nurse Innovator, Children's National

Lori Irvin | NICU RN and Nurse Innovator | Children's National » speaking at Orphan Drug Congress

Dwain Irvin, Chief Executive Officer, NovAccess Global, Inc

Dwain Irvin | Chief Executive Officer | NovAccess Global, Inc » speaking at Orphan Drug Congress

Stephen Kagan, Global Medical Lead, Genetic Medicine and Pipeline, Rare Disease, Pfizer

Stephen Kagan | Global Medical Lead, Genetic Medicine and Pipeline, Rare Disease | Pfizer » speaking at Orphan Drug Congress

Judith Kelleher-Andersson, President and Chief Executive Officer-Neuronascent, NeuroNascent Inc

Judith Kelleher-Andersson | President and Chief Executive Officer-Neuronascent | NeuroNascent Inc » 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

Ameet Khara, Head, Patient Advocacy, Scholar Rock

Ameet Khara | Head, Patient Advocacy | Scholar Rock » speaking at Orphan Drug Congress

Mark Kiel, Chief Scientific Officer, Genomenon

Mark Kiel | Chief Scientific Officer | Genomenon » speaking at Orphan Drug Congress

Maria Kirsch, General Manager, Patient Services, EVERSANA

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

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

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

Yishai Knobel, CEO, RxWare

Yishai Knobel | CEO | RxWare » speaking at Orphan Drug Congress

David Knouft, Senior Vice President, Market Access and Field Commercial, Harmony Biosciences, LLC

David Knouft | Senior Vice President, Market Access and Field Commercial | Harmony Biosciences, LLC » speaking at Orphan Drug Congress

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

Akihiro Ko | Chief Executive Officer | Elixirgen Therapeutics, Inc. » speaking at Orphan Drug Congress

Bryan Kobel, Chief Executive Officer, TC Biopharm

Bryan Kobel | Chief Executive Officer | TC Biopharm » speaking at Orphan Drug Congress

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, Principal, IPM.ai

Ivan Kouchlev | Principal | IPM.ai » speaking at Orphan Drug Congress

Kollet Koulianos, Senior Payer/Provider Consultant, The National Hemophilia Foundation

Kollet Koulianos | Senior Payer/Provider Consultant | The National Hemophilia Foundation » 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

Henrik Krook, Vice President Of Commercial Operations, Egetis Therapeutics AB

Henrik Krook | Vice President Of Commercial Operations | Egetis Therapeutics AB » speaking at Orphan Drug Congress

Sanath Kumar Ramesh, Chief Executive Officer, OpenTreatments Foundation

Sanath Kumar Ramesh | Chief Executive Officer | OpenTreatments Foundation » 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

Cath Lawrence, VP, Rare Disease, Bionical Emas

Cath Lawrence | VP, Rare Disease | Bionical Emas » speaking at Orphan Drug Congress

Del Lebel, Head, US Government Affairs and Policy, Alexion – AstraZeneca Rare Disease

Del Lebel | Head, US Government Affairs and Policy | Alexion – AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Nirosha Lederer, Head, US Government, Aetion

Nirosha Lederer | Head, US Government | Aetion » speaking at Orphan Drug Congress

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

Connie Lee | President And Chief Executive Officer | Alliance to Cure Cavernous Malformations » speaking at Orphan Drug Congress

Jeremy Levin, Chairman and Chief Executive Officer, Chairman, BIO, Ovid Therapeutics

Jeremy Levin | Chairman and Chief Executive Officer, Chairman, BIO | Ovid Therapeutics » speaking at Orphan Drug Congress

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

Jeff Livingstone, Chief Executive Officer, Igia Pharmaceuticals

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

John Lo, Chief Commercial Officer, Beam Therapeutics

John Lo | Chief Commercial Officer | Beam Therapeutics » speaking at Orphan Drug Congress

Brett Logan, Director of US Market Access & State Government Relations, Alliance for Regenerative Medicine

Brett Logan | Director of US Market Access & State Government Relations | Alliance for Regenerative Medicine » speaking at Orphan Drug Congress

Robert Long, Executive Director, Uplifting Athletes

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

Michael Lynch, Head of Regulatory Affairs CMC, Regulatory Operations and Regulatory Operational Excellence, Alexion – AstraZeneca Rare Disease

Michael Lynch | Head of Regulatory Affairs CMC, Regulatory Operations and Regulatory Operational Excellence | Alexion – AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Richa Madan Lomash, Scientific Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS, NIH

Richa Madan Lomash | Scientific Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS | NIH » speaking at Orphan Drug Congress

Amanda Malakoff, Executive Director, Rare Disease Company Coalition

Amanda Malakoff | Executive Director | Rare Disease Company Coalition » speaking at Orphan Drug Congress

Manoj Malhotra, Chief Medical Officer, Ovid Therapeutics

Manoj Malhotra | Chief Medical Officer | Ovid Therapeutics » 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

Silas Martin, Senior Director, Market Access Scientific And External Strategy, Johnson & Johnson

Silas Martin | Senior Director, Market Access Scientific And External Strategy | Johnson & Johnson » speaking at Orphan Drug Congress

Paolo Martini, Chief Scientific Officer Rare Diseases, Hematology and External R&D, Moderna, Inc.

Paolo Martini | Chief Scientific Officer Rare Diseases, Hematology and External R&D | Moderna, Inc. » speaking at Orphan Drug Congress

Kristin Marvin-Keller, Managing Director, H4B-Boston

Kristin Marvin-Keller | Managing Director | H4B-Boston » speaking at Orphan Drug Congress

An Massaro, Lead Neonatologist, Neonatology Program, Office of Pediatric Therapeutics, FDA

An Massaro | Lead Neonatologist, Neonatology Program, Office of Pediatric Therapeutics | FDA » speaking at Orphan Drug Congress

Annette Maughan, Co-Founder And Chief Executive Officer, KBG Foundation

Annette Maughan | Co-Founder And Chief Executive Officer | KBG Foundation » speaking at Orphan Drug Congress

Cara Mayfield, VP, Patient Advocacy and Corporate Communications, Homology Medicines

Cara Mayfield | VP, Patient Advocacy and Corporate Communications | Homology Medicines » speaking at Orphan Drug Congress

Maria Mccaffrey, Vice President Country Regulatory and Quality, Alexion – AstraZeneca Rare Disease

Maria Mccaffrey | Vice President Country Regulatory and Quality | Alexion – AstraZeneca Rare Disease » speaking at Orphan Drug Congress

Cate McCanless, Vice President Of External Affairs, Harmony Biosciences, LLC

Cate McCanless | Vice President Of External Affairs | Harmony Biosciences, LLC » speaking at Orphan Drug Congress

Reenie McCarthy, Chief Executive Officer, Stealth BioTherapeutics

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

Kim McClellan, President, Recurrent Respiratory Papillomatosis Foundation

Kim McClellan | President | Recurrent Respiratory Papillomatosis Foundation » speaking at Orphan Drug Congress

Susan Mccune, Vice President, Pediatrics & Clinical Pharmacology, thermo fisher scientific

Susan Mccune | Vice President, Pediatrics & Clinical Pharmacology | thermo fisher scientific » speaking at Orphan Drug Congress

Kristin McKay, Executive Director, Project Alive -

Kristin McKay | Executive Director | Project Alive - » speaking at Orphan Drug Congress

John Mckew, CSO, Lumos Pharma

John Mckew | CSO | Lumos Pharma » speaking at Orphan Drug Congress

Jennifer McNary, Founder, One Rare

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

Christine McSherry, Executive Director, Strategic Collaborations, The Emmes Company, LLC

Christine McSherry | Executive Director, Strategic Collaborations | The Emmes Company, LLC » speaking at Orphan Drug Congress

Zdravka Medarova, Founder, Chief Technology Officer, TransCode Therapeutics

Zdravka Medarova | Founder, Chief Technology Officer | TransCode Therapeutics » speaking at Orphan Drug Congress

Jen Melanson, Patient Advocate and Caregiver, Rare Disease

Jen Melanson | Patient Advocate and Caregiver | Rare Disease » speaking at Orphan Drug Congress

Giancarlo Mennella, Managing Director, Egamid Ltd.

Giancarlo Mennella | Managing Director | Egamid Ltd. » speaking at Orphan Drug Congress

Ann-Lise Mikolajczak, Manager Global Health and Access, IFPMA

Ann-Lise Mikolajczak | Manager Global Health and Access | IFPMA » 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

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

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

Mara Molinello, Senior Vice President, Head of US Commercial, Alnylam Pharmaceuticals

Mara Molinello | Senior Vice President, Head of US Commercial | Alnylam Pharmaceuticals » speaking at Orphan Drug Congress

Mona Moonis, NA Brand Lead, Rare Disease, Kyowa Kirin

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

Joyce Moore, Executive Director, Patient Recruitment Strategies, Allucent

Joyce Moore | Executive Director, Patient Recruitment Strategies | Allucent » speaking at Orphan Drug Congress

Kimberly Moran, Head of Rare Diseases, US, UCB

Kimberly Moran | Head of Rare Diseases, US | UCB » speaking at Orphan Drug Congress

Meghan Morgan-Smith, Associate Director of Therapeutic Expertise Center for Rare Diseases, ICON

Meghan Morgan-Smith | Associate Director of Therapeutic Expertise Center for Rare Diseases | ICON » speaking at Orphan Drug Congress

Maria-Cruz Morillo, TA Head of Rare Disease, Allucent

Maria-Cruz Morillo | TA Head of Rare Disease | Allucent » speaking at Orphan Drug Congress

Mary Morlino, Patient Services Manager, Global Genes

Mary Morlino | Patient Services Manager | Global Genes » speaking at Orphan Drug Congress

Claire Murphy, General Manager - Life Sciences US, Terrapinn

Claire Murphy | General Manager - Life Sciences US | Terrapinn » speaking at Orphan Drug Congress

Joseph Musumeci, President, BluePrint Orphan

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

Michael Nace, Brand Director, Rare Disease Advisor

Michael Nace | Brand Director | Rare Disease Advisor » 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

George Naumov, Chief Business Officer, RS Oncology

George Naumov | Chief Business Officer | RS Oncology » speaking at Orphan Drug Congress

Adora Ndu, Chief Regulatory and interim Legal Officer, BridgeBio Gene Therapy

Adora Ndu | Chief Regulatory and interim Legal Officer | BridgeBio Gene Therapy » speaking at Orphan Drug Congress

Catherine Nester, Senior Vice President HCP and Patient Engagement, Inozyme Pharma

Catherine Nester | Senior Vice President HCP and Patient Engagement | Inozyme Pharma » speaking at Orphan Drug Congress

Galina Nesterova, Executive Medical Director- Rare Diseases & Pediatrics, PPD, part of Thermo Fisher Scientific

Galina Nesterova | Executive Medical Director- Rare Diseases & Pediatrics | PPD, part of Thermo Fisher Scientific » speaking at Orphan Drug Congress

Victoria Niklas, Chief Medical Officer, Oak Hill Bio

Victoria Niklas | Chief Medical Officer | Oak Hill Bio » speaking at Orphan Drug Congress

Megan O'Boyle, Patient Engagement Lead, Global Genes, RARE-X

Megan O'Boyle | Patient Engagement Lead, Global Genes | RARE-X » speaking at Orphan Drug Congress

Phillip Okwo, Patient, Sickle Beta Zero Thalassemia

Phillip Okwo | Patient | Sickle Beta Zero Thalassemia » speaking at Orphan Drug Congress

Curtis Oltmans, Chief Legal Officer, Fulcrum

Curtis Oltmans | Chief Legal Officer | Fulcrum » speaking at Orphan Drug Congress

Elizabeth Ottinger, Project Management Group Leader, NIH/NCATS

Elizabeth Ottinger | Project Management Group Leader | NIH/NCATS » speaking at Orphan Drug Congress

Lisa Padilla, Chief Executive Officer, Beryl Therapeutics Inc

Lisa Padilla | Chief Executive Officer | Beryl Therapeutics Inc » speaking at Orphan Drug Congress

Jennifer Panagoulias, Chief Regulatory and Compliance Officer, Mahzi Therapeutics

Jennifer Panagoulias | Chief Regulatory and Compliance Officer | Mahzi Therapeutics » speaking at Orphan Drug Congress

Maria Pandolfo, Senior Vice President, Patient Services, Catalyst Pharmaceutical Partners

Maria Pandolfo | Senior Vice President, Patient Services | Catalyst Pharmaceutical Partners » speaking at Orphan Drug Congress

Kinnari Patel, President & COO, Rocket Pharma

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

Alana Paull, Director of Associate, Genentech

Alana Paull | Director of Associate | Genentech » 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

Marissa Penrod, Founder and CEO, Rare Disease Advocate, Mom to son with Duchenne Muscular Dystrophy, Team Joseph

Marissa Penrod | Founder and CEO, Rare Disease Advocate, Mom to son with Duchenne Muscular Dystrophy | Team Joseph » speaking at Orphan Drug Congress

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

Sue Peschin | President and Chief Executive Officer | Alliance for Aging Research » speaking at Orphan Drug Congress

Steven Peskin, President and Chief Executive Officer, Formerly Executive Medical Director Population Health at Horizon Blue Cross Blue Shield o, SRP Advisors, LLC

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

Wyndi Phillips, VP, Project Management, WEP Clinical

Wyndi Phillips | VP, Project Management | WEP Clinical » 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

Peter Pitts | President | C.M.P.I » speaking at Orphan Drug Congress

Tobias Polak, Director Real-World Data, myTomorrows

Tobias Polak | Director Real-World Data | myTomorrows » speaking at Orphan Drug Congress

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

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

Stuart Portman, Health Policy Advisor, United States Senate Committee On Finance

Stuart Portman | Health Policy Advisor | United States Senate Committee On Finance » speaking at Orphan Drug Congress

Joshua Prabhakar, Vice President, Novartis Gene Therapies

Joshua Prabhakar | Vice President | Novartis Gene Therapies » speaking at Orphan Drug Congress

Damon Race, President & Chief Executive Officer, GeneVentiv Therapeutics, Inc

Damon Race | President & Chief Executive Officer | GeneVentiv Therapeutics, Inc » speaking at Orphan Drug Congress

Rachel Radomski, Senior Director & Head, Patient Advocacy, Harmony Biosciences

Rachel Radomski | Senior Director & Head, Patient Advocacy | Harmony Biosciences » speaking at Orphan Drug Congress

Paula Ragan, President and Chief Executive Officer, X4 Pharmaceuticals

Paula Ragan | President and Chief Executive Officer | X4 Pharmaceuticals » speaking at Orphan Drug Congress

Harpreet Ram, Senior Advisor and Subject Matter Expert, GARDaccess,, Partnership for Quality Medical Donations (PQMD)

Harpreet Ram | Senior Advisor and Subject Matter Expert, GARDaccess, | Partnership for Quality Medical Donations (PQMD) » speaking at Orphan Drug Congress

Amy Raymond, Senior Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials

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

Debra Regier, Chief, Genetics and Metabolism, Children's National Hospital

Debra Regier | Chief, Genetics and Metabolism | Children's National Hospital » speaking at Orphan Drug Congress

Frank Rivera, President, Stronger Than Sarcoidosis

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

Noreen Rizvi, Principal, Third Rock Ventures

Noreen Rizvi | Principal | Third Rock Ventures » speaking at Orphan Drug Congress

Derek Robertson, President, Maryland Sickle Cell Disease Association

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

Priscilla Rodriguez, Diversity Inclusion Advocacy Manager, EveryLife Foundation for Rare Diseases

Priscilla Rodriguez | Diversity Inclusion Advocacy Manager | EveryLife Foundation for Rare Diseases » speaking at Orphan Drug Congress

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

Heidi Ross | Vice President of Policy and Regulatory Affairs | National Organization for Rare Disorders » speaking at Orphan Drug Congress

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

Jean-Pascal Roussy | Head, Global Public Affairs, Rare Diseases and Rare Blood Disorders | Sanofi » speaking at Orphan Drug Congress

Kacper Rucinski, Delegate, SMA Europe / Founder, Member of Strategy Board, SMA Foundation Poland, SMA Europe (Germany) / SMA Foundation (Poland)

Kacper Rucinski | Delegate, SMA Europe / Founder, Member of Strategy Board, SMA Foundation Poland | SMA Europe (Germany) / SMA Foundation (Poland) » speaking at Orphan Drug Congress

Christopher Rudolf, Chief Executive Officer and Founder, volv global

Christopher Rudolf | Chief Executive Officer and Founder | volv global » speaking at Orphan Drug Congress

Pierantonio Russo, Chief Medical Officer, Data and Analytics,, EVERSANA

Pierantonio Russo | Chief Medical Officer, Data and Analytics, | EVERSANA » speaking at Orphan Drug Congress

Kareem Saad, Chief Transformation Officer, GeneDx

Kareem Saad | Chief Transformation Officer | GeneDx » 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

Sandhya Sanduja, Branch Chief, Pharmacology/Toxicology Branch 2, Office of Therapeutic Products, FDA

Sandhya Sanduja | Branch Chief, Pharmacology/Toxicology Branch 2, Office of Therapeutic Products | FDA » speaking at Orphan Drug Congress

Sergio Santillana, Chief Medical Officer, Ikena Oncology

Sergio Santillana | Chief Medical Officer | Ikena Oncology » speaking at Orphan Drug Congress

Aaron Schuchart, CBO, Lumos Pharma

Aaron Schuchart | CBO | Lumos Pharma » speaking at Orphan Drug Congress

Jonathan Schwartz, Chief Gene Therapy Officer, Rocket Pharma

Jonathan Schwartz | Chief Gene Therapy Officer | Rocket Pharma » speaking at Orphan Drug Congress

Denise Scots Knight, Chief Executive Officer, Mereo Biopharma

Denise Scots Knight | Chief Executive Officer | Mereo Biopharma » speaking at Orphan Drug Congress

Simone Seiter, Senior Partner, Simon-Kucher

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

Albert Seymour, President & Chief Executive Officer, Homology Medicines

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

Christy Shafer, Chief Commercial Officer, Marinus Pharmaceuticals

Christy Shafer | Chief Commercial Officer | Marinus Pharmaceuticals » speaking at Orphan Drug Congress

Nilomi Shah, Director of Medical Affairs Lead, Lexeo Therapeutics

Nilomi Shah | Director of Medical Affairs Lead | Lexeo Therapeutics » speaking at Orphan Drug Congress

Pallavi Sharma, Senior Director, Scientific Program Management, Adverum Biotechnologies

Pallavi Sharma | Senior Director, Scientific Program Management | Adverum Biotechnologies » speaking at Orphan Drug Congress

Conor Sheehey, Health Policy Advisor, United States Senate

Conor Sheehey | Health Policy Advisor | United States Senate » speaking at Orphan Drug Congress

Jerry Shen, Chief Executive Officer, Co-Founder, AceLink Therapeutics, Inc

Jerry Shen | Chief Executive Officer, Co-Founder | AceLink Therapeutics, Inc » 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

Wen Chyi Shyu, Chief Executive Officer, BRIM Biotechnology, Inc.

Wen Chyi Shyu | Chief Executive Officer | BRIM Biotechnology, Inc. » speaking at Orphan Drug Congress

Sean Sigmon, VP Business Development, InformedDNA

Sean Sigmon | VP Business Development | InformedDNA » speaking at Orphan Drug Congress

Dylan Simon, Director of Policy, EveryLife Foundation for Rare Diseases

Dylan Simon | Director of Policy | EveryLife Foundation for Rare Diseases » speaking at Orphan Drug Congress

Amy Simon, Chief Medical Officer, Beam Therapeutics

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

Amanda Singleton, Director, Biopharma and Strategic Partnerships, GeneDx

Amanda Singleton | Director, Biopharma and Strategic Partnerships | GeneDx » 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

Tom Smith, Strategic Initiatives Director, Durbin

Tom Smith | Strategic Initiatives Director | Durbin » speaking at Orphan Drug Congress

Rorey Smith, Community Member, TREND Community

Rorey Smith | Community Member | TREND Community » speaking at Orphan Drug Congress

Rachel Smith, Executive Director of Rare Disease, Parexel International

Rachel Smith | Executive Director of Rare Disease | Parexel International » speaking at Orphan Drug Congress

Kim Smith-Whitley, Site Lead, Advisor, Scientific and Clinical Affairs, Pfizer

Kim Smith-Whitley | Site Lead, Advisor, Scientific and Clinical Affairs | Pfizer » speaking at Orphan Drug Congress

Scott Snyder, Chief Digital Officer, EVERSANA

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

Marcio Souza, President and Chief Executive Officer, Praxis Precision Medicines

Marcio Souza | President and Chief Executive Officer | Praxis Precision Medicines » speaking at Orphan Drug Congress

Rodica Stan, Senior Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS, NIH/NCATS

Rodica Stan | Senior Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS | NIH/NCATS » 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, MLD Foundation

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

Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases

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

Marshall Summar, Director, Rare Disease Institute, Children's National Medical Center

Marshall Summar | Director, Rare Disease Institute | Children's National Medical Center » speaking at Orphan Drug Congress

Jason Tardio, Chief Operating Officer, Ovid Therapeutics

Jason Tardio | Chief Operating Officer | Ovid Therapeutics » speaking at Orphan Drug Congress

Mitra Tavakkoli, Vice President; Clinical Development, 4DMT

Mitra Tavakkoli | Vice President; Clinical Development | 4DMT » 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

Corinne TRUFFAULT, INTSEL CHIMOS - CEO, EUROPACCESS

Corinne TRUFFAULT | INTSEL CHIMOS - CEO | EUROPACCESS » 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

Lori Upham, HIE Program Leader, ReAlta Life Sciences

Lori Upham | HIE Program Leader | ReAlta Life Sciences » speaking at Orphan Drug Congress

Jennifer Urwongse, DCT Strategy & Innovation Director, PPD, part of Thermo Fisher Scientific

Jennifer Urwongse | DCT Strategy & Innovation Director | PPD, part of Thermo Fisher Scientific » speaking at Orphan Drug Congress

Vinnie Ustach, Applied Computational Biology Lead, GeneDx

Vinnie Ustach | Applied Computational Biology Lead | GeneDx » speaking at Orphan Drug Congress

Ben van der Schaaf, Partner, Arthur D. Little

Ben van der Schaaf | Partner | Arthur D. Little » speaking at Orphan Drug Congress

Robert-Jan Van Son, General Manager Germany, BeNeLux and Nordic,, Orphalan

Robert-Jan Van Son | General Manager Germany, BeNeLux and Nordic, | Orphalan » 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

Atul Varadhachary, Managing Partner, Fannin Innovation Studio

Atul Varadhachary | Managing Partner | Fannin Innovation Studio » speaking at Orphan Drug Congress

Lavni Varyani, Founder, Pharma BP DMCC

Lavni Varyani | Founder | Pharma BP DMCC » speaking at Orphan Drug Congress

Geeta Vemuri, Managing Partner, Agent Capital

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

Ernesto Vera Sanchez, VP, Dept. Head GXP Strategy, RDC, Allucent

Ernesto Vera Sanchez | VP, Dept. Head GXP Strategy, RDC | Allucent » 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

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

Terence Walts, Chief Executive Officer, Cambium Medical Technologies

Terence Walts | Chief Executive Officer | Cambium Medical Technologies » speaking at Orphan Drug Congress

Mary Wang, Programme Director, Rare Diseases International

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

Jingsong Wang, President & Chief Executive Officer, Harbour Biomed

Jingsong Wang | President & Chief Executive Officer | Harbour Biomed » speaking at Orphan Drug Congress

Amanda Wang, Vice President Oncology Precision Medicine Franchise Lead For Immuno-Oncology, Bayer Pharma AG

Amanda Wang | Vice President Oncology Precision Medicine Franchise Lead For Immuno-Oncology | Bayer Pharma AG » speaking at Orphan Drug Congress

Laurent WARTELLE, INTSEL CHIMOS – Business Development Manager, EUROPACCESS

Laurent WARTELLE | INTSEL CHIMOS – Business Development Manager | EUROPACCESS » speaking at Orphan Drug Congress

Yael Weiss, Chief Executive Officer, Mahzi Therapeutics

Yael Weiss | Chief Executive Officer | Mahzi Therapeutics » speaking at Orphan Drug Congress

Steve Winitsky, Vice President, Technical,, Parexel

Steve Winitsky | Vice President, Technical, | Parexel » speaking at Orphan Drug Congress

Ryan Witt, Chief Business Officer, Spinogenix

Ryan Witt | Chief Business Officer | Spinogenix » 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, CEO & Chair, Canadian Organisation for Rare Diseases & Rare Diseases International

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

Teonna Woolford, Chief Executive Officer, Sickle Cell Reproductive Health Education Directive

Teonna Woolford | Chief Executive Officer | Sickle Cell Reproductive Health Education Directive » speaking at Orphan Drug Congress

Chris Wurst, Director, Business Development & Sales Operations, WEP Clinical

Chris Wurst | Director, Business Development & Sales Operations | WEP Clinical » speaking at Orphan Drug Congress

Lisa Yañez, Chief Executive Officer, Aerami Therapeutics

Lisa Yañez | Chief Executive Officer | Aerami Therapeutics » speaking at Orphan Drug Congress

Yuliya Yasinskaya, Medical Officer, FDA

Yuliya Yasinskaya | Medical Officer | FDA » 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

Jennifer Young, Vice President, Global Policy and Public Affairs - Specialty Care, Pfizer

Jennifer Young | Vice President, Global Policy and Public Affairs - Specialty Care | Pfizer » speaking at Orphan Drug Congress

Christine Yu, Chief Executive Officer, P2P Syncro

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

Marjorie Zettler, Executive Director of Clinical Science, Regor Pharmaceuticals Inc.

Marjorie Zettler | Executive Director of Clinical Science | Regor Pharmaceuticals Inc. » speaking at Orphan Drug Congress

Tara Ziegler, Business Development Manager, GeneDx

Tara Ziegler | Business Development Manager | GeneDx » speaking at Orphan Drug Congress

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