2024 Speakers
Suchismita Acharya, Chief Executive Officer, AyuVis Research
Samuel Agyei Wiafe, Executive Director, Rare Disease Ghana Initiative
Dennis Akkaya, Chief Commercial Officer, myTomorrows
David Allison, Chief Executive Officer, TREAT-NMD
Derek Ansel, Executive Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials
Irina Antonijevic, Chief Medical Officer, EveryONE Medicines
Victoria Arteaga, Co-founder, SHER Hispanic Society for Rare Diseases
Ethan Ash, Senior VP of Business Development, BioNews
Kevin Bagley, Former Medicaid Director for State of Nebraska | Consultant, Kevin Bagley Strategic Consulting
Mark Bainbridge, Senior Director, Business Development, Early Access, Inceptua Group
Ritu Baral, Managing Director And Senior Biotechnology Analyst, Cowen
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
Kevin Baruzzi, Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH
Stuart Bell, Senior Vice President, Inceptua Early Access, Inceptua Group
Jana Benesh, Director of Therapeutic Expertise, ICON Centre for Rare Diseases
Kara Berasi, Chief Executive Officer, Haystack Project
Alexandre Bétourné, Executive Director, Critical Path Institute
Sulagna Bhattacharya, Chief Executive Officer, Nanoscope Therapeutics
Robin Bliss, VP Strategic Consultant, Veristat
Lara Bloom, President and CEO, The Ehlers-Danlos Society
Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research
Morgan Boname, Senior Director, Public Policy, Neurocrine Biosciences
Natasha Bonhomme, Founder, Expecting Health
James Boomhower MS(c), FP-C, NR-P, CCISM, Code Rare EMS Educator, Rare Strides
Wendy Borsari, Sr. Manager of Patient Advocacy, Tenaya Therapeutics
Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
P.J. BROOKS, Deputy Director of the NCATS Division of Rare Diseases Research Innovation, National Institutes of Health
Benjamin Brown, Executive Director, American Society of Pharmacovigilance
Bert Bruce, US Rare Disease Lead, Pfizer, Pfizer
Chuck Bucklar, Principal, Chair 5 Consulting, LLC
Karin Butler, Head of Technical Research, York Health Economics Consortium
Lauren Bylsma, Senior Epidemiologist, EpidStrategies, A Division of ToxStrategies
Amanda Cali, Trustee,The Radiant Hope Foundation; Trustee,Ian Cali FOP Research Fund at University of PA; Co-Founder/Officer, Tin Soldiers Global
Robert M. Califf, Commissioner, U.S. Food and Drug Administration
Joshua Card-Gowers, Senior Evidence Lead, HealthLumen
Madeleine Carrier, Patient Navigator, myTomorrows
Geoffrey Case, Digital Editor, RARE Revolution Magazine
Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi
James Chambers, Associate Professor, Tufts Medical Center
Chere Chapman, Chief Executive Officer, Ardea Outcomes
Richard Chapman, Chief Science Officer, Innovation and Value Initiative
Ellen Cho, Director, Specialty Programs, Direct Relief
Priya S Chockalingam, Vice President, Head of Clinical Bioanalytics & Translational Sciences, Beam Therapeutics
Duane Clark, General Manager, US Rare Disease, SANOFI
Kyle Clifton, Senior Director, Red Nucleus
Jason Colquitt, Chief Executive Officer, Across Healthcare
Matt Conway, Vice President of Strategic Initiatives, Rare Strides
Colin Cook, Technical Founder, XDemics Corporation
Ron Cooper, Former CEO, Albireo Pharma
Donna Cowan, Associate Director EAP, Stealth BioTherapeutics
Carole Cramer, President & Founder, BioStrategies
Joslyn Crowe, Executive Director, National Niemann Pick Disease Foundation Inc
Andrew Cummins, VP Access and Reimbursement, Sciensus
Colleen Dansereau, Senior Director - Gene Therapy Program and Office of Emerging Medical Discoveries, Boston Children's Hospital
Zayd Daruwala, VP, Medical Strategy, Havas Life Rare
Michelle Davis, Executive Director, International Fibrodysplasia Ossificans Progressiva (FOP) Association, Inc.
Khrystal K Davis, Founding President, Texas Rare Alliance
Marcella Debidda, President, BioNews Clinical, BioNews Clinical
Daniel DeFabio, Rare Dad (Menkes Disease), Co-Founder, Disorder: The Rare Disease Film Festival
Tom Defay, Deputy Head Diagnostics Strategy & Development, Alexion Pharmaceuticals
Linda Dell, VP, Program Leader, ReAlta Life Sciences, Inc.
Jin Ding, Research Fellow, University of Sheffield
Kate Donigan, Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs, Sarepta Therapeutics
Lauren Dougherty, Chief Operating Officer, TREND Community
Kasey Edwards, Co-Founder, Community Relations Specialist, Cure AP-4
Leslie Edwin, President, Cushing's Support & Research Foundation
Michael Eging, Executive Director, Rare Access Action Project
Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals
Jida El Hajjar, Executive Director,, ALS Action Canada
Alexis Elder, Board Member, Cure VCP Disease
Shaun Ellis, Vice President, Global Market Access Specialty Ophthalmology, Johnson & Johnson
Terri Ellsworth, Patient Advocate Partnerships, myTomorrows
Bastiaan Evers, Senior Director Drug Discovery, Scenic Biotech
Erik Feingold, CEO & Co-Founder, FDNA, Inc
James Fink, Director, Epilepsy Therapeutic Lead, Quiver Bioscience
Alicia Fiscus, Head of Global Regulatory Affairs and QA, Rhythm Pharmaceuticals
Colton Flowers, NLP Engineer, TREND Community
Pawel Fludzinski, Chief Executive Officer, AmideBio
Max Flurie, Senior Data Scientist, TREND Community
Benjamin Forred, Director of Translational Research and the CoRDS Registry, Sanford Health
Lindy Forte, Senior Vice President, Market Access and Pricing, EVERSANA
Jean-Pascal Fournier-Roussy, Head, Global Public Affairs, Rare Diseases & Rare Blood Disorders, Sanofi
Nick France, Vice President, Clinical Development, Neurology, Alexion, AstraZeneca Rare Disease
Sebastian Franzinger, Head of Business Development, Scenic Biotech
Albert Freedman, Counseling Psychologist / Rare Dad, Rarecounseling.com
Jon Fryzek, Practice Director, EpidStrategies, A Division of ToxStrategies
Mike Fusakio, Lead Consultant, Regulatory Affairs, Halloran Consulting Group
Renata Gallagher, MD, PhD, Medical Director, Parexel
Neha Gandhi, Consultant, Advocacy and Policy
Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Sarah Gheuens, Chief Medical Officer, Head of R&D, Agios Pharmaceuticals
Diego Gil Cardozo, President, Enfermedades Raras en el Caribe y América Latina (ERCAL)
Christoph Glaetzer, Chief Global Value and Access Officer, Johnson & Johnson
Sarah Glass, Chief Operating Officer, n-Lorem Foundation
Josie Godfrey, Co-Founder and Chief Executive Officer, Realise Advocacy
Leslie Gordon, Medical Director, Progeria Research Foundation
Deb Gouveia, SVP, Development Operations, EveryONE Medicines
Heidi Grabenstatter, Science Director, Critical Path Institute
Nathan Grant, Researcher, Medical Student; President and Founder, Harvard Medical School; Siblings with a Mission
Amy Gray, CEO, Undiagnosed Diseases Network Foundation
Brandi Greenberg, VP Marketing-Analytics & Insights, Real Chemistry
James Griffin, Sickle Cell Patient Advocate, Independent Milwaukee Wisconsin
Gay Grossman, Co-Founder and Patient Advocate, ADCY5.org
Teri Hagemann, Director of Client Services, Splash Clinical
Alaa Hamed, Global Head, Medical Affairs Rare Disease and Rare Blood Disorders, Sanofi
Matthew Hanchard, Research Fellow, University of Sheffield
Kate Hanman, US Head of Rare Diseases, Costello Medical
Rachel Harrison, Pre-Approval Access Lead, argenx
Sue Harvey, Founder & Chief Transformation Officer, Riverwise Leadership
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics
Alexandra Heumber Perry, Chief Executive Officer, Rare Diseases International
Gary Ho, Senior Community Director, TREND Community
Lauren Holder, Producer/Host, Help 4 HD Live, Help 4 HD
Laura Hopkins, Associate Director, Critical Path Institute
Spencer Huggett, Founder, Synapze
Wills Hughes-Wilson, Chief Patient Access & Commercial Planning, Mereo Biopharma
Huong Huynh, Director of Regulatory Science, Critical Path Institute
Oxana Iliach, Member of Regulatory Science Committee, International Rare Diseases Research Consortium (IRDiRC)
Katheron Intson, Chief Executive Officer, Varient
Roberto Iunes, Senior Health Economist, World Bank
Sun-Gou Ji, Vice President of Computational Genetics, BridgeBio Pharma
Gretchen Jordan, Associate Director, Cushing's Support & Research Foundation
Lisa Jordan, Senior Vice President, Management Supervisor, Havas Life Rare
Andria Joseph, Project Director Outcomes Research, York Health Economics Consortium
Christine Juday, Head, Market Access, Real Chemistry
Richie Kahn, Co-Founder & Principal, Canary Advisors
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Inderpreet Kambo, Principal (Partner) - Information Management & Analytics, IQVIA
Manohar Katakam, Chief Executive Officer, SteroTherapeutics
Ken Kengatharan, Managing General Partner, Atheneos Ventures
Melissa Kennedy, Chief Executive Officer, International Rett Syndrome Foundation
Annie Kennedy, Chief of Policy, Advocacy, & Patient Engagement, EveryLife Foundation for Rare Diseases
Adrian Kielhorn, Senior Director Health Economics and Outcomes Research, Alexion
Amy Kimzey, Toxicologist, EpidStrategies, A Division of ToxStrategies
MARIA KIRSCH, President, Patient Services, EVERSANA
Terri Klein, President & Chief Executive Officer, National M.P.S. Society
Jessica Klein, Rare Disease Awareness Marketer, KnoJAK Consulting
Aki Ko, Chief Executive Officer, Elixirgen Therapeutics, Inc.
Bret Koncak, Co-Founder, mejo
Lauren Kopsick, Founder and Executive Director, The Healthcare Navigation Project
Augusta Elizabeth Koroma, Chief Executive Officer, Sickle cell Intervention U.K
Ivan Kouchlev, Managing Principal, Real-World Data Solutions, Real Chemistry
Rahul Kukreja, Project Director, Clinical Project Management, Allucent
Neha Kumar, Global Commercial Strategy - Precision Medicine, Johnson & Johnson Innovative Medicine
Alix Lacoste, AI Computational Biology Director Genetic Insights, Invitae
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
Larissa Lapteva, Associate Director, FDA
Sherry Lard, PhD, Associate Director for Product Management (ADPM), U.S. Food and Drug Administration
Patroski Lawson, Chief Executive Officer, The KPM Group DC
Yann Le Cam, Founder and past-CEO, EURORDIS
Connie Lee, President and Chief Executive Officer, Alliance to Cure Cavernous Malformations
Michael Lehmicke, Senior Vice President of Science and Industry Affairs, Alliance for Regenerative Medicine
Melanie Lendnal, Senior Vice President, Policy and Advocacy, The ALS Association
Maurice Leonard, Medical Director, Uniphar
William Lin, Program Director, GARDaccess, Partnership for Quality Medical Donations (PQMD)
Andrew Lo, Charles E. and Susan T. Harris Professor, Massachusetts Institute of Technology (MIT)
Robert Long, Executive Director, Uplifting Athletes
Veronica Lopez Gousset, MPH, Founder and President, VLG Global
Cathleen Lutz, Vice President of the Rare Disease Translational Center, Jackson Laboratory
Gary M Hutchinson, SVP, Cold Chain Engineering, Modality Solutions
Kim MacDonnell, Associate Director, Rare Disease, COE, Parexel
Donna Mackey, Vice President, Clinical Operations, Akouos, Inc.
Mark Manfredi, Chief Executive Officer, Ikena Oncology
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Silas Martin, Head, Access & Policy Research, Johnson & Johnson
Craig Martin, President, Rithm Health; CEO, Orphan Therapeutics Accelerator
Jaclyn Martin, Vice President of Global Head Market Access Cell and Gene Therapy, Bayer
Kristin Marvin-Keller, Managing Director, Havas Life Rare
Sweety Mathew, Senior Regulatory Professional, Novo Nordisk
Annette Maughan, CEO/Co-Founder, KBG Foundation
Gemma Mayman, Specialty Care Patient Solutions and Engagement Lead, Pfizer
Reenie McCarthy, Chief Executive Officer, Stealth BioTherapeutics
Alice McConnell, Chief Executive Officer, Speragen, Inc
Michael McGinn, Founder, CureLBSL
Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research
Gina McHugh, Conference Producer, Terrapinn
Kristin McKay, President/Executive Director, Project Alive
Jennifer McNary, Founder, One Rare
Stuart Mealing, Associate Director (HEOR), York Health Economics Consortium
Laurence Mignon, Executive Director, Clinical Development, n-Lorem Foundation
Sheila Mikhail, Executive Director, Columbus Children's Foundation
Tsveta Milanova, Chief Commercial Officer, Agios Pharmaceuticals
Nicole Miller, Vice President, Molecular Diagnostics, Global Medical Affairs, Ultragenyx
Nicola Miller, Editor in Chief, RARE Revolution Magazine
Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease, Worldwide Clinical Trials
Samarendra Mohanty, Founder-President, Chief Scientific Officer, Nanoscope Therapeutics
James Mongiardo, Chief Executive Officer, A-Synaptic Corp.
Mary Morlino, Patient Navigator, Undiagnosed Disease Network Foundation (UDNF)
Lindsay Murphy, Senior Group Director, Integrated Intelligence, Real Chemistry
Joe Musumeci, President, BluePrint Orphan
Ramaiah Muthyala, Professor/Director at University of Minnesota, President and Chief Executive Officer,, Indian organization for Rare diseases
Ramaa Nathan, VP of Data Science and Real-World Evidence, EVERSANA
Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Neena Nizar, Assoc. Director, Patient Advocacy Strategy, ICON Centre for Rare Diseases; President, The Jansens Foundation
Greg Palko, Vice President, North America Oncology Region Franchise Head, Kyowa Kirin
Jim Palma, Chief Executive Officer, TargetCancer Foundation
Effie Parks, Podcast Host, Director of Parnerships, Once Upon A Gene - CTNNB1
Kinnari Patel, President & COO, Rocket Pharma
Dave Pearce, Chair, International Rare Diseases Research Consortium (IRDiRC)
Melissa Penn, Director Patient Engagement Research & Development, Bayer
Sue Peschin, President and Chief Executive Officer, Alliance for Aging Research
Steven Peskin, President and Chief Executive Officer; Former Executive Medical Director Population Health, Horizon Blue Cross Blue Shield of NJ, SRP Advisors, LLC
Maria Picone, Chief Executive Officer, TREND Community
Armelle Pindon, Chief Scientific Officer, Cure VCP Disease
Peter Pitts, President, C.M.P.I
Christopher Porter, Vice President, Government Affairs and Policy, Travere Therapeutics
Otto Prohaska, Founder and Chief Executive Officer, Transcytos
XENIA PROTON DE LA CHAPELLE, Chief Executive Officer and Patient Representative, AtmosR
Harsha Rajasimha, Founder and Chairman, IndoUSrare
Harpreet Ram, President, EVR Consulting
Dorian Readnour, Executive Director, Global Patient Access, Sarepta Therapeutics
Debra Regier, Director, Rare Disease Institute, Children's National Hospital
Deborah Requesens, Director, JumpStart, Orphan Disease Center, University of Pennsylvania, Perelman School of Medicine
Joshua Resnikoff, Chief Executive Officer, TMA precision health
Jamie Ring, Founder, Jamie Ring Advocacy Consulting
Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries
Frank Rivera, President, Stronger Than Sarcoidosis
Afshan Rizvi Hussain, Managing Director, Global Rare Disease Lead, Havas Health
Brigitte Roberts, Director, Program Management, Comcast
Derek Robertson, President, Maryland Sickle Cell Disease Association
Christopher Robertson, Professor of Law, Boston University
Devin Rosenthal, Vice President & Head of Due Diligence, NovaQuest Capital Management
Heidi Ross, Vice President of Policy and Regulatory Affairs, National Organization for Rare Disorders
Ruth Rostron, Director of Program Management, Uniphar
Ronenn Roubenoff, Consultant, Ronenn Roubenoff
Yury Rozenman, SVP of Business Development Life Sciences, Ada Health
Christopher Rudolf, Founder and Chief Executive Officer, volv global
Pierantonio Russo, FCCP, FAAP, STS, Corporate Chief Medical Officer, EVERSANA
Naomi Sacks, Principal Scientist, HEORStrategies, A Division of ToxStrategies LLC
Michael Salgaller, Supervisor, Technology Analysis and Marketing Unit (TAMU), National Institute of Health (NIH)
Matt Salo, Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer, Salo Health Strategies
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Dick Salvatierra, President, Americas Health Foundation
Rachael Sawaya, Associate Director, Project Management – Execution, PCM Trials
Anthony Scatamacchia, Senior Vice President, Innovation, Patient Services, Eversana
Mary Schaheen, President, Prevail Partners Llc
Charles Schmidt, Senior Medical Advisor, iNSTITUTO JÔ CLEMENTE
Matthias P. Schönermark, M.D., Ph., Managing Director, SKC Beratungsgesellschaft mbH
Carolyn Schwartz, President and Chief Scientist, DeltaQuest Foundation
Denise Scots Knight, Chief Executive Officer, Mereo Biopharma
Simone Seiter, Senior Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH
Albert Seymour, President & Chief Executive Officer, Homology Medicines
Amit Sharma, Vice President Clinical Development - Global Therapeutic Area Head Nephrology & Hematology, Alexion Pharmaceuticals
Ryan Sheedy, Founder, mejo
Darlene Shelton, President/Founder, Danny’s Dose Alliance
Michael Sherman, MD, MBA, MS, Venture Partner, RA Capital Management
Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research
John Sholar, Chief Executive Officer, jCyte Inc
Mridula Shukla, Head of Regulatory Affairs, Gene Therapy, Poseida Therapeutics, Inc.
Amanda Singleton, Biopharma Partnerships, GeneDx
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Rachel Smith, Global Head, Rare Disease, Parexel
Laura Smith van Carroll, Head of Insight & Advocacy, Metabolic Support UK
Oskar Smrzka, Chief Executive Officer/CSO, Ablevia
Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics
Rodica Stan, Senior Project Manager, NIH/NCATS
Paul Stanton, Senior Director, Global Strategy, Early Access, Inceptua Group
Kim Stephens, Executive Director, Muenzer MPS Research & Treatment Center
Dean Suhr, President and co-Founder, MLD Foundation
Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases
Donna Sullivan, Director Of Patient Advocacy, Pathways To Trust
Marshall Summar, Chief Executive Officer, Uncommon Cures
Laura Tadvalkar, Managing Director, RA Capital Management
Julia Taravella, Exececutive Director, Rare Trait Hope Fund
Jason Tardio, Chief Operating Officer, Ovid Therapeutics
Matt Teuteberg, President/ CEO, Splash Clinical
Pete Thornton, VP, Pharma Services & Strategic Partnerships, Invitae
Maria Törnsén, President, North America, Calliditas Therapeutics
Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center
Anthy Tsatoumas, Manager, Project Management, Veristat
Andrew Udell, President of North America, Commercial, Calliditas Therapeutics
Nicholas Valle, Senior Product Director, iTakeControl, Red Nucleus
Jytte van Huijstee, Director Clinical Trial Operations, myTomorrows
Leon van Wouwe, Clinical Innovation Director, volv global
Lavni Varyani, Founder, Pharma Business Partners
Geeta Vemuri, Managing Partner, Agent Capital
Raghu Vishwanath, Global Head, Franchise Strategy and Operations, Rare Diseases Specialty Care, SANOFI
Lindsey Wahlstrom-Edwards, Partnerships Lead, Sano Genetics
Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio
Mary Wang, Programme Director, Rare Diseases International
Albert Wang, Co-founder, CEO, Comend
Jon Weber, Senior Director of Cell & Gene Therapies Access Lead, Bayer
Michael Weickert, Chief Executive Officer, Pacylex Pharmaceuticals
Patricia Weltin, Chief Executive Officer, Beyond the Diagnosis
Melissa Williams, Founder, Simply Patient
Ben Willis, Director of Business Excellence, Egetis Therapeutics
Richard Wilson, SVP North America Rare Disease Franchise Head, Kyowa Kirin
Kasey Woleben, Founder, Cure Mito Foundation/Rare Village
Kristin Viswanathan Wolff, Senior Director, Global Government Affairs And Public Policy, bluebird bio
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
Justin Yang, Co-Founder, Kaibab Health & DMV Bio
Christine Yu, Chief Executive Officer, P2P Syncro
Samantha Zappia, MS, RAC, Principal Consultant, SK Regulatory Solutions
Sophia Zilber, Board member, patient registry director, Cure Mito Foundation
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