2022 Speakers
Dennis Akkaya, Chief Commercial Officer, myTomorrows
Faraz Ali, Chief Executive Officer, Tenaya Therapeutics
Paul Aliu, Head Global Governance Office, Novartis Chief Medical Office (CMO), Novartis
Simon Andrews, Senior Vice President, Head of Analytics, EVERSANA
Samer Ansari, Head of Data, Digital & Technology, Takeda Oncology, Takeda
Derek Ansel, Senior Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials
Catherine Argyriou, Postdoctoral Fellow, McGill University
Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy, National Institute for Health and Disability Insurance
Lisa Bacon, Senior Director, Enzyvant Therapeutics
Ella Balasa, Rare Disease, Patient Advocate
Peter Bannister, VP, Life Sciences, Ada Health
Julie Batista, Director of Epidemiology and Biostatistics, Sanofi Genzyme
Katherine Beaverson, Executive Director and Head of Patient Advocacy and External Engagement, Rare Disease Research Unit,, Pfizer
Stuart Bell, Vice President, Consulting, Inceptua
Joseph Bender, Head of Global Rare Disease Registries, Sanofi Genzyme
Chris Benevides, Associate Director, Strategic Solutions, Elligo Health Research
Alon Ben-Noon, Chief Executive Officer, NeuroSense Therapeutics
Wendy Benson, Chief Strategy and Innovation Officer, Rady Children's Institute for Genomic Medicine
Allyson Berent, Chief Science Officer, Foundation for Angelman Therapeutics, COO and Co-Founder, GeneTx Biotherapeutics
Scott Bergeron, Partner, Clinical Research, iTakeControl, a Red Nucleus Company
Terry Jo Bichell, Founder And Director, COMBINEDBrain
Gus Bilirakis, Rare Disease Caucus Lead, United States Congress
Nadia Bodkin, OLM DEI Expert, Rare Advocacy Movement
Nicole Boice, Founder & Executive Director, RARE-X
Natasha Bonhomme, Chief Strategy Officer, Genetic Alliance Founder, Expecting Health
Simone Boselli, Director, Public Affairs, EURORDIS
Jason Bradt, Executive Director of Clinical Research, Pharming Healthcare
Larry Bressler, Head of Value and Market Access, Chiesi Global Rare Diseases
Phillip Brooks, Acting Director, Office of Rare Diseases Research, NIH - NCATS
Amy Brower, US National Director, Newborn Screening Translational Research Network (NBSTRN),, American College of Medical Genetics and Genomics
Robert M. Califf, Commissioner, U.S. Food and Drug Administration
Jason Cameron, Chief Operating Officer, Orphan Drug Consulting
Katherine Capperella, VP of Global Patient Engagement, Office of the Chief Medical Officer,, Janssen Pharmaceutical companies of Johnson & Johnson
Elena Cardenal, Scientific Director, Dravet Syndrome Foundation Spain
Nicole Carswell, Executive Director, Patient Engagement and Recruitment,, Premier Research
Grant Castor, Vice President Commercial Operations, Sentynl Therapeutics
Jessica CerulloMerrill, Vice President, VOZ Advisors
Anne-Sophie Chalandon, Global Rare Disease Policy Head, Sanofi Genzyme
Corin Chapman, Director, GFPD Board of Directors
Lynsey Chediak, Head of Partnerships, Rare Base
Melissa Coloton, EVP, Business Development and Marketing, Scout Clinical
Jason Colquitt, Chief Executive Officer, Across Healthcare
Michele de Guise, Scientific Vice-President, INESSS
Tom Defay, Deputy Head Diagnostics Strategy & Development, Alexion Pharmaceuticals
Francisco Diaz-Mitoma, CMO, VBI Vaccines
Chowdary Dondapati, Executive Director, US Head of Diagnostic Marketing, Precision Medicine, BAYER
Danielle Dong, Global Operations and Advocacy Lead, Rare Disease Registries, Sanofi Genzyme
Robert Donnell, Executive Vice President, Medicines Access, Smartway Pharma
Marcus Droege, Vice President, Global Value & Market Access, Passage Bio
Stephanie Duffy, Global Patient Advocacy and Engagement Director, The Janssen Pharmaceutical Company of Johnson and Johnson
Sanjoy Dutta, Chief Scientific Officer, JDRF
Sean Ekins, Founder And Chief Executive Officer, Collaborations Pharmaceuticals
Matthew Ellinwood, Chief Scientific Officer, National MPS Society
Terri Ellsworth, Consultant, myTomorrows
Izaskun Elorza, Senior Medical Director,, Parexel
Wendy Erler, Vice President, Global Head Patient Experience & Insights, Alexion AstraZeneca Rare Disease
Jorge Faccinetti, Chairman and Chief Executive Officer, Pituitary World News
Lynn Fallon, President, CTI Clinical Trial and Consulting
Qadan Farah, Clinical Pharmacist Researcher, Curant Health
Sarah Farr, Associate, Genesys Capital Partners
Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc
James Fink, Senior Principal Scientist, Q-State Biosciences
Aliza Fink, Director of Research Programs, National Organization for Rare Disorders (NORD)
Ryan Fischer, Chief Advocacy Officer, Parent Project Muscular Dystrophy
Daniel Fischer, President and Chief Executive Officer, Tevard Biosciences
Al Freedman, Counseling Psychologist, Freedman Counseling Associates
Pat Furlong, Founding President And Chief Executive Officer, Parent Project Muscular Dystrophy
Milda Galkute, Researcher, Spanish Federation of Rare Disease
Nicolas Garnier, Senior Director Patient Advocacy Lead, Pfizer
Safiyya Gassman, Senior Director, Policy & Public Affairs, Rare Disease & Gene Therapy, Pfizer
Joseph Geraci, Chief Scientific Officer, Nurosene Health Inc.
Lev Gerlovin, Vice President, Life Sciences Practice, Charles River Associates
Diego Fernando Gil Cardozo, Executive Director, Federación Colombiana de Enfermedades Raras -FECOER-
Christoph Glaetzer, Chief Global Value and Access Officer, Janssen
Sarah Glass, Chief Development Officer, n-Lorem Foundation
Richard Gliklich, Chief Executive Officer, OM1
Deb Gouveia, SVP Development Operations, Triplet Therapeutics
Scott Gray, Chief Executive Officer, Clincierge
Fumie Griego, Deputy Director General And Chief Operating Officer, International Federation of Pharmaceutical Manufacturers and Associations
Edith Gross, Health and Social Science Senior Project Manager, EURORDIS
Abhi Gupta, Senior Director, Global Gene Therapy, Pfizer
Pamela Guthrie, Director, Sales and Marketing, Clincierge
Elin Haf Davies, Chief Executive Officer, Aparito
Sam Hall, Managing Partner, Undisclosed Venture Fund
Michael Hamper, Medical/Science Advisory, Kleine-Levin Syndrome Foundation
Christopher Hamper, Medical Advisory Board Member, Kleine-Levin Syndrome Foundation
Guido Hartmann, Head of Translational Pharmacology, Centogene Switzerland AG
Scott Harwood, Director, Check Rare
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics
Rachele Hendricks-Sturrup, Research Director, Real World Evidence, Duke Margolis Center for Health Policy
Matthew Hewitt, Executive Director, Scientific Services Cell and Gene Therapy, Charles River Laboratories
Claudia Hirawat, Owner & Executive Chair, VOZ Advisors
Juliet Hulse, Senior Director, Global RN Strategy and Patient Advocacy, Illingworth Research
Gwen Hyland, Head of Commercial Excellence, Rare Diseases, AstraZeneca, Alexion Pharmaceuticals
Hearn Jay Cho, Chief Medical Officer, Multiple Myeloma Research Foundation
Padmini Jayaraman, Director, Corporate and Business Development, Laekna Therapeuics
Chris Jones, Partner, Clinical Research, iTakeControl, a Red Nucleus Company
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Rashida Karmali, Chief Executive Officer, Tactical Therapeutics Inc
Vikram Karnani, Executive Vice President, President, International, Horizon Therapeutics
Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical
Lindsay Kehoe, Project Manager, Clinical Trials Transformation Initiative (CTTI)
Ken Kengatharan, Managing General Partner, Atheneos Ventures
Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement,, EveryLife Foundation for Rare Diseases
Doug Kerr, Chief Medial Officer, Generation Bio
Kelly Kester, Director, Advanced Therapy Solutions, Cardinal Health
Kritika Khurana, Account Executive, Commercial Team, AllStripes
Maria Kirsch, General Manager, Patient Services, EVERSANA
Lise Kjems, Chief Medical Officer, Cyclo Therapeutics, Inc.
Matthew Klein, Chief Operating Officer, PTC therapeutics
Walter Kowtoniuk, Venture Partner, Third Rock Ventures, LLC
Sarka Kubinova, Medical evaluator, MoCA coordinator, Section of Pricing and Reimbursement Regulation, Czech Republic State Institute for Drug Control
Harsh Kulkarni, Consultant, ZS
Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics
Janet Lynch Lambert, Chief Executive Officer, Alliance for Regenerative Medicine
David Lapidus, President, Lapidus Data
Catherine Lawrence, Head of Rare Disease, Bionical Emas
Yann Le Cam, Chief Executive Officer, EURORDIS
Kimberly LeBlanc, Director, UDN Coordinating Center, Harvard Medical School, Harvard Medical School
Seth Lederman, Co-Founder, Chief Executive Officer & Chairman, Tonix Pharmaceuticals
Naomi Litchfield, Global Patient Advocacy Lead, Bionical Emas
Jeff Livingstone, Chief Executive Officer, Igia Pharmaceuticals
Sam Lucas, SVP- Expanded Access Programs, Durbin- part of Uniphar Group
Sanjeev Luther, President & Chief Executive Officer, Cornerstone Pharmaceuticals
Wolfram Lux, Senior Director, Market Access & Commercial Strategy, Rare Diseases,, Simon-Kucher & Partners
Flaminia Macchia, Executive Director, Rare Diseases International
Geoff Mackay, President And Chief Executive Officer, AVROBIO
Natalia Maeva, President, The Bulgarian Society of Patients with Pulmonary Hypertension
Nitin Mahadev, Vice President, Global Franchise Lead, Rare Disease, Pfizer
Will Maier, Vice President, Rare Disease, Drug Development Sciences, ICON plc
Nick Marchant, Patient & Health Impact lead, Rare Disease, Pfizer
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Michael Marquez, Nanoscope Therapeutics
Craig Martin, Chief Executive Officer, Global Genes
Brad Martin, Vice President, Market Access, Taysha Gene Therapies
Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Therapeutics
Liz Mascherino, Associate Director of Patient Engagement, Equity and Transparency, Biogen
Carie Masoner, Executive Director, Regulatory & Scientific Affairs, CTI Clinical Trial and Consulting
Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research
Kelly McKee, Vice President, Patient Registries and Recruitment, Medidata
Jennifer McNary, Founder, One Rare
Christine Mcsherry, Chief Executive Officer, Casimir Trials
Wesley Michael, President, Rare Patient Voice, LLC
Christeen Moburg, Vice President Patient Advocacy & Government Relations, Sangamo Therapeutics
Samarendra Mohanty, Founder-President, Chief Scientific Officer, Nanoscope Therapeutics
Mona Moonis, NA Brand Lead, Rare Disease, KKI
Mark Moore, CEO, GemPharmatech US
Maria-Cruz Morillo, Global Therapeutic Operations Lead, Allucent
Christopher Moxham, Chief Scientific Officer, Rarebase, PBC
Andrew Mumford, Managing Director, Initiate
Michael Murphy, Chief Medical & Scientific Officer, Worldwide Clinical Trials
Joseph Musumeci, President, BluePrint Orphan
Paul Nash, Senior Director, Simon-Kucher & Partners
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Indu Navar, Founder and Chief Executive Officer, EverythingALS
Amy Nicole Nayar, VP US Patient Advocacy and Government Affairs, Novartis Gene Therapies
Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD)
Robert Neusner, Director, Global Commercial Strategy,, Janssen Pharmaceutical companies of Johnson & Johnson
Amanda Nickles Fader, Professor of Gynecology and Obstetrics, Professor of Oncology, Vice-Chair, Gynecologic Surgical Operations,, Johns Hopkins Medical Center
Kris O’Brien, Executive Director, Program Strategy, Rare Disease and Pediatrics, Premier Research
Andrew Obenshain, Chief Executive Officer, Bluebird Bio
Matan Ofir, Director, Medison Ventures
Cristol O'Loughlin, Founder & Chief Executive Officer, ANGEL AID CARES
Asif Paker, VP Clinical Development, SwanBio Therapeutics
Jim Palma, Executive Director, TargetCancer Foundation
Francis Pang, VP Global Market Access, Orchard Therapeutics
Lisa Parker, VP, Medical, emotive
Kinnari Patel, President & COO, Rocket Pharma
Bhavesh Patel, Principal, CRA, Charles River Associates
Luciana Peixoto, Member of Parents Advisory Board, Systemic JIA Foundation
Melissa Penn, Director Patient Engagement Research & Development, Bayer
Maria Picone, CEO, TREND Community
Richa Poddar, Chief Commercial Officer, Agios Pharmaceuticals
Ana Puhl, Senior Research Scientist, Collaborations Pharmaceuticals
Pam Rattananont, Director, Customer Strategy and Innovation, Parexel
Shannon Resetich, Global Franchise Head, Rare Diseases, Sanofi Genzyme
Michael Rice, Senior VP, Head of Advanced Therapeutics, Lumanity
David Ringger, Head of Market Access, Initiate
David Rose, Business Development, Rare Revolution
Luke Rosen, Founder, KIF1A.ORG
Johanna Rossell, Chief Commercial Officer, Enzyvant
Ruth Rostron, Director of Program Management, Durbin- part of Uniphar Group
Christopher Rudolf, CEO, volv global
Ashish Sagrolikar, Chief Operating Officer, Zogenix Inc.
Dharmendra Sahay, ZS
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Denise Schulz, Associate Director of Clinical Operations,, Horizon Therapeutics
Simone Seiter, Partner, Simon-Kucher & Partners
Antonino Severino, Head CTS, Nextpharma
Gopi Shanker, Chief Scientific Officer, Tevard Biosciences
Panna Sharma, Chief Executive Officer, President and Director, Lantern Pharma
Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research
Fanny Sie, One Roche, Head of Artificial Intelligence and Digital Health, Roche
Stuart Siedman, Head of Patient Advocacy, Chiesi Global Rare Disease
Amy Simon, Chief Medical Officer, Beam Therapeutics
Jaswinder Singh Khera, Managing Director, WEP Clinical
Nick Sireau, Chair and Chief Executive Officer, AKU Society
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Charlie Small, Associate Director, Integrated Trial Solutions, Elligo Health Research
Todd Smith, President, Chief Commercial Officer, Tolmar Pharmaceuticals Inc
Scott Snyder, Chief Digital Officer, EVERSANA
Charlene Son Rigby, Chief Executive Officer, RARE-X
Matt Sowards, Chief Innovation Officer, Scout Clinical
Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products, Medicines Evaluation Board, Medicines Evaluation Board
Swami Subramanian, Head, US/Global Pricing and Contracting, Regeneron
Dean Suhr, President, MLD Foundation
Jamie Sullivan, Director of Public Policy, EveryLife Foundation
Noreen Sullivan, Senior Principal, Value & Development Consulting, Evidera PPD
Web Sun, Co-Founder & President, Komodo Health
Lee Sutton, Vice President, Business Development, WEP Clinical
Tim Szymusiak, Head of Market Access Services, BluePrint Orphan
Mitra Tavakkoli, VP, Clinical Development, -
Amy Teets, Global Public Affairs Head, Pompe and MPS, Sanofi Genzyme
Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences
Michael Thomas, Principal, ZS
Kate Tighe, Head, US Public Affairs & Patient Advocacy - Rare Diseases, Sanofi Genzyme
Mark Trusheim, Strategic Director, MIT NEWDIGS
Andrew Udell, President of North America, Commercial, Calliditas Therapeutics
Fyodor Urnov, Scientific Director, Innovative Genomics Institute, Professor, Department of Molecular & Cell Biology, University of California, Berkeley
Zizi Uzezi Imatorbhebhe, Senior Vice President, Global Strategy and Development, Head Rare Disease Innovation Center,, Ergomed
Henri Vaitkevicius, VP of Clinical Development,, Marinus Pharmaceuticals
Kristin Van Goor, Lead, North America Regulatory Policy, Vertex Pharmaceuticals
Suneet Varma, Global President, Rare Disease Business Unit, Pfizer inc
Geeta Vemuri, Managing Partner, Agent Capital
Richard Vesely, Vice President, Regulatory Strategy,, Allucent
Erin Wade, Content Specialist, myTomorrows
Mary Wang, Science Policy Manager, Rare Diseases International
Dan Wasserstrom, Senior Director, Business Development, Clinigen Group
Christopher Watson, Director, Consulting, Thread Research
Kevin Weatherwax, Vice President, Head of Regulatory Affairs, WEP Clinical
Robert Weinstein, Division of Infectious Diseases, Rush University
Ellen Weiss, Vice President, In-Home Solutions, Decentralized Clinical Trials, PCM Trials
Gareth Williams, President - Sciensus Rare, Sciensus
Toni Williams, Global Business Development Director, Clinigen Group
Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Disorders
Richard Xie, Health Economist & Director of Research, The Innovation and Value Initiative IVI
Rachel Yang, Secretary, Council Member, RDI, Head of International Affairs, Chinese Alliance for Rare Diseases (CHARD)
David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.
Christine Yu, Chief Executive Officer, P2P Syncro
Nancy Yu, Co-Founder & Chief Executive Officer, AllStripes Research
Martine Zimmermann, Global Head Of Regulatory Affairs, Alexion Pharmaceuticals
Shiran Zimri, Head of Scientific Program, NeuroSense Therapeutics
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