2025 Speakers
Mackenzie Abramson, MPH, BCPA, Patient Advocate & Sr. Manager, Community Engagement and Communications, Global Genes
Dean Aguiar, Vice President, Translational Research, TSC Alliance
Lynn Albizo, Chief Public Policy Officer, Immune Deficiency Foundation
Kathryn Alexander, Consultant, SMS Mom & Advocate
Amanda Almany, Parent Advisor, NKH Crusaders
Ahmad Alrawi, SVP Corporate Strategy, Commense Bio
Kristin Archibald, Founder, NKH Crusaders
Barbara Arone, VP, Global Category Lead, Evidence Generation for Medical Affairs, IQVIA
Ethan Ash, Senior VP of Business Development, Bionews
Alithea Athans, Patient Columnist, CAD
Leone Atkinson, Executive Medical Director, Rare Disease, Advanced Therapies and Pediatrics Team, Fortrea
Lisa Bair, Managing Director, Market Access and Payer Marketing, BGB Group
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
Oliver Barnes, US Pharmaceutical & Biotech Correspondent, Financial Times
Erica Barnes, Rare Disease Advisory Council Executive Director, University of Minnesota
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
Thomas Bartlett, Rare Disease Patient Digital Technology Advisor, Bartlett Rare Disease Advocacy
Kevin Baruzzi, Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH
Alison Bateman-House, Assistant Professor, Division of Medical Ethics, NYU Grossman School of Medicine
Andree Bates, Chief Executive Officer, Eularis Ltd
Scott Baver, VP, Head of US Medical Affairs, ITF Therapeutics
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
Anny Bedard, President, Egetis Therapeutics US
Hourinaz Behesti, Founder & CEO, Hebbian Bio
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
Michelle Boisvert, PhD Student, Harvard Medical School
Julieta B Bonvin Sallago, Lead Clinical Research Associate/ Assistant Professor, Connecticut Children's/ University of Connecticut School of medicine
Ian Bonzani, Sr. Principal, Real World Technology Solutions, IQVIA
Rohit Borker, Senior Vice President, Value Access, Health Economics & Outcomes Research, RTI Health Solutions
Lesa Brackbill, Associate Director of Advocacy, Patient Advocacy Strategies
Teresa Brandt, VP, Regulatory Affairs, Avidity Biosciences
Christine Brennan, Managing Director, Vertex Ventures HC
Jonathan Bruce, Vice President, Market Access, Distribution, HEOR, F2G, Inc.
Mariana Burin, Medical Director, Thermo Fisher Scientific
Raquel Cabo, Former SVP Corporate Affairs and Commercial strategy, Ovid Therapeutics
Melody Calkins, Director, Healthcare Policy, Biotechnology Innovation Organization
Mary Callanan, Senior Director, Global Medical Affairs, Alnylam Pharmaceuticals
Mindy Cameron, President, AdvocacyWorks Consulting
Tanisha Carino, Visiting Fellow, Duke-Margolis Institute for Health Policy
Janet Carlson, CEO, 1.11 Group
Joel Cartner, Director, Access Policy, Muscular Dystrophy Association
Michelle Carty, Senior Principal, Patient Centered Solutions, IQVIA
Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience
Jill Castle, Director of Patient Education & Patient Advocacy, Little Hercules Foundation
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
Chelsea Catsburg, Director, BluePrint Orphan
James Chambers, Professor, Tufts Medical Center
Hayley Chapman, Senior Program Director, Patient Focused Drug Development
Timothy Chen, Medical Director, Caliway Biopharmaceuticals
Ellen Cho, Senior Director, Specialty Programs, Direct Relief
Duane Clark, General Manager, US Rare Disease, Sanofi
Jim Coccia, Partner, TKG Access
Jason Colquitt, Chief Executive Officer, Across Healthcare
Amy Comstock Rick, Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA
Rena Conti, Associate Professor, Markets, Public Policy, and Law, Boston University
Donna Cowan, Director EAP, Stealth BioTherapeutics
Michael Cristello, Director, Marketing, Rare Disease, Sumitomo Pharma
Eric Crombez, MD, EVP, Chief Medical Officer, Ultragenyx
Glenna Crooks, PhD, Founder and CEO, Strategic Health Policy International, Inc
Lucy Culp, Vice President, State Government Affairs, The Leukemia & Lymphoma Society (LLS)
Andrew Cummins, VP Access and Reimbursement, Sciensus
Colleen Dansereau, RN, Vice President - Emerging Discoveries, Boston Children's Hospital
Marcella Debidda, President Patient Insights & Clinical Solutions, BioNews Clinical
Brigid DeCoursey Bondoc, Partner, Morrison Foerster
Daniel DeFabio, Global Genes Director of Education & Co-Founder,, Disorder: The Rare Disease Film Festival
Marcus Delatte, VP, Regulatory Strategy, Allucent
Rumi Desai, Director, Global Regulatory Strategy, Amgen, Inc.
Veruska di Sena, SVP, Head of US Medical Affairs, Intercept Pharmaceuticals, Inc.
Bradley Dickerson, VP of Project Management & Patient Advocacy, IECURE Inc
Elizabeth Donahue, Senior Director, Project Management, Thermo Fisher Scientific
Danielle Dong, Global Medical Scientific Advocacy Lead, Rare Disease, Sanofi
Sika Dunyoh, Senior Director of Patient Advocacy, Travere Therapeutics
Heidi Edwards, President & Founder, Sisters’ Hope Foundation
Erika Edwards, Health and Medical Reporter and Writer, NBC News
Michael Eging, Executive Director, Rare Access Action Project
Jill Elliott, Vice President, Marketing, X4 Pharmaceuticals
Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics
Eric Feigen, Regulatory Affairs Manager, Immune Deficiency Foundation
Anthony Fiori, Senior Managing Director, Manatt
Alicia Fiscus, Head of Global Regulatory Affairs and QA, Rhythm Pharmaceuticals
Amber Fisher, Senior Clinical Research Manager, Kura Oncology Inc
E'Lissa Flores, Director, Science & Regulatory Affairs, Biotechnology Innovation Organization
Amanda Forys, Managing Partner, Magnolia Market Access
Pangkong Fox, Science Engagement Director, CACNA1A Foundation, Inc.
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
Suzanne Gaglianone, Patient Care Field Representative, ReveraGen BioPharma Inc
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)
Victoria Gemme, Director of Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)
Stephen George, PharmD, MS, SVP Market Access Reimbursment, Milliman
Nayan Ghosh, Head of Life Sciences & HealthTech, Marwood Group
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
Federico Goodsaid, SVP Regulatory and Quality, Ariana Pharma
Vivek Gowda, Head of Research, Neoclease
J. Michael Graglia, Co-Founder & Managing Director, Syngap Research Fund
James Griffin, Sickle Cell Patient Advocate, Independent Milwaukee Wisconsin
Kurt Gunter, Clinical Development Advisor, Steminent Biotherapeutics Inc.
David Gusick, Executive Director, Somebody To Talk To
Joern-Peter Halle, Advisor and Venture Partner, BioGeneration Ventures (BGV)
Sonit Handa, Member, Board of Directors, The Josselyn Center
Kate Hanman, US Head of Rare Diseases, Costello Medical
Rachel Harrison, Director, Expanded Access Program, Argenx
Thomas Hart, Director of Outreach, Critical Path Institute
Christina Hartman, Senior Director, Government Affairs, Orchard Therapeutics
Scott Harwood, Director, Check Rare
Atalah Haun, MD, Vice President, Medical Affairs, WEP Clinical
Guadalupe Hayes Mota, CEO & Co-founder, Stealth Star-up & Senior Lecturer, Massachusetts Institute of Technology
Matthew Hayes, FSA, MAAA, Principal and Consulting Actuary, Milliman
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics
Brandon Michael Henry, Medical Director, Cell and Gene Therapy, IQVIA
Michele Herndon, Program Director, UDNF
Anne Heyes, Vice President, Head of Value Strategy, RTI Health Solutions
Lauren Holder, Producer/Host, Help 4 HD Live Podcast, Help 4 HD
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
Mohammad Imran, Global BD Head, 3H Medi Solution Inc
Neil Inhaber, Head, Rare Diseases, Global Medical Affairs, Takeda
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
Puja Joshi, Medical Director, Clinical Development, Stoke Therapeutics
Richie Kahn, Co-Founder & COO, Canary Advisors
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Litsa Kapsalis, PhD student, Massachusetts Institute of Technology
Tina Karunaratne, Founder and CEO, Karuna Integrated Clinical Services
Mwango Kashoki, MD, MPH, SVP, Global Head of Regulatory Strategy, Parexel
Joe Katakowski, Director of Research, RTW Foundation
Mandeep Kaur, MD, MS, Board Member, Former SVP Horizon / Amgen
Louise Kearney, Executive Director, Rare disease, Advanced therapies and Pediatrics Team (RAPT), Fortrea
Tara Keith, Freelance writer, Rare disease advisor and SM companion
Jessica Klein, Patient Advocacy & Industry Relations, Senior Liaison – East Region, Acadia Pharmaceuticals Inc.
Lauren Kopsick, Founder & Executive Director, The Healthcare Navigation Project
Jonathan Kornstein, VP, Rare Disease & Pediatrics Clinical Development, Caidya
Graeme Ladds, CEO & Owner, PharSafer
Lukas Lange, Chief Executive Officer & Co-Founder, Probably Genetic
Patroski Lawson, Co-founder, The Just Society
Kimberly LeBlanc, Director, UDN Data Management Coordinating Center, Harvard Medical School, Harvard Medical School
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
Michael Lingzhi Li, Assistant Professor of Business Administration, Harvard Business School, Technology and Operations Management
Dan Lier, Advocate/Patient/Speaker, Somebody To Talk To
Naomi Litchfield, Director Patient Advocacy, Bionical Emas
Andrew Lo, Charles E. and Susan T. Harris Professor, MIT Sloan School of Management
Mathieu Loiseau, Director of Rare Clinical Services, Sciensus
Rob Long, Executive Director, Uplifting Athletes
Veronica Lopez Gousset, MPH, Steering Committee Member, HTAi Rare Disease Interest Group
Peng Lu, Chief Medical Officer, Pharvaris
Vera Luxner, Director of Advocacy Relations, Rare Disease Advisor
Larry Luxner, -, -
Alastair MacDonald, Vice President, Medical Affairs, Sciensus
Kim MacDonnell, Associate Director, Rare Disease, Parexel
Victor Maertens, Government Affairs Director, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Alexandra Mangili, Clinical Science Head, Alfasigma
Maureen Mansfield, Vice-Chair, ENET and Founder, Protect Your Passion
Yuri Maricich, MD, MBA;, Chief Medical Officer, CAMP4 Therapeutics
Silas Martin, Head, Access & Policy Research, Johnson & Johnson
Craig Martin, Founder, CEO, Orphan Therapeutics Accelerator
Kristin Marvin-Keller, Managing Director, Havas Life Rare
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
Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research
Jennifer McNary, Co-Founder, Canary Advisors
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
Mary Morlino, Patient Navigator, Undiagnosed Disease Network Foundation (UDNF)
Rachit Mukkamala, President, MIT Biotech Group, Massachusetts Institute of Technology
Joe Musumeci, President, BluePrint Orphan
Ramaiah Muthyala, Professor/Director at University of Minnesota, President and Chief Executive Officer,, Indian organization for Rare diseases
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, Senior Conference Director, Terrapinn
Katie Niemeyer, Speaker, Philanthropist, CRNA, SJS Survivor, Katie Niemeyer, Speaker LLC
Kevan Oconnor, Venture Investor, Cystic Fibrosis Foundation
Eunhee Ohgami, CEO, NeuroCores, Inc.
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
Alexandre Pare, Scientific Researcher, McGill University
Effie Parks, Podcast Host, CTNNB1 Connect & Cure, Once Upon A Gene - CTNNB1
Simrit Parmar, Founder, cellenkos
Kinnari Patel, President, Head of R&D and COO, Rocket Pharma
Doug Paul, VP Commercial Strategy, Cypress Biopharma
Dave Pearce, Chair, International Rare Diseases Research Consortium (IRDiRC)
Allison Peck, Co-Foundet, Cure VCP Disease
Melissa Penn, Director Patient Engagement Research & Development, Bayer
Mike Porath, Founder, Executive Director, Dup15q Alliance, The Mighty
Bryce Powerman, Director, Business Development, Natera
Matthew Prentice, Director of State Policy, Immune Deficiency Foundation
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
Patrick Raletz, Project Manager, Bionical Emas
Harpreet Ram, President, EVR Consulting
Dhaval Rathod, Associate Director, Pharm Sci, Shionogi Inc
Dorian Readnour, Vice President, International, Sarepta Therapeutics
Sabrina Rebello, Senior Program Manager, Amyloidosis Research Consortium
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
Sumira Riaz, Health Psychologist & Patient Engagement Consultant, Unboxed Psychology
Faisal Riaz, Medical Director Neuroimmunology and AATD, Takeda Pharmaceuticals USA Inc
Sara Riordan, Vice President, Strategy, Genome Medical
Frank Rivera, President, Stronger Than Sarcoidosis
Luis Rojas, Chief Clinical Development Officer, Polaryx Therapeutics
Hector Rosado, Director, Business Strategy & Operations, Takeda
Luke Rosen, Founder, Rescue 7: Firefighters for Patients
Johanna Rossell, Senior Vice President, General Manager, Sumitomo Pharma
Seth Rotberg, Senior Manager, Patient Advocacy, Solid Biosciences
Jean-Pascal Roussy, Head, Global Public Affairs, Rare Diseases, Sanofi
Christian Rubio, Executive Director, EverythingALS
Christopher Rudolf, Founder and Chief Executive Officer, Volv Global
Pierantonio Russo, FCCP, FAAP, STS, Corporate Chief Medical Officer, EVERSANA
Matt Salo, CEO, Salo Health Strategies
Marina Sardone, Global Head of Patient Advocacy, Alfasigma
William Sarraille, Adjunct Professor, University of Maryland Francis King Carey School of Law
Mary Schaheen, President, Prevail Partners Llc
Jeremy Schmahmann, Founding Director, Massachusetts General Hospital Ataxia Center, Massachusetts General Hospital Ataxia Center
Jörg Schüttrumpf, Chief Scientific Innovation Officer, Grifols
Carolyn Schwartz, President & Chief Scientist, DeltaQuest Foundation
Jessica Scott, Chief Executive Officer, Legacy Health Strategies
Josephine Scrofani, Customer Strategy Lead, Bayer Pharmaceuticals
Simone Seiter, Senior Partner, Simon-Kucher & Partners Strategy & Marketing Consultants GmbH
Kathy Senecal, Volunteer, Rare Disease Patient
Seungoh Seo, Senior AI Researcher, ONCOCROSS
Bridget Seritt, Founder, Advocates for Compassionate Therapy Now
Andrew Shao, SVP of Global Scientific and Regulatory Affairs, Niagen Bioscience Inc
Jordan Shin, Chief Medical Officer, HAYA Therapeutics
Tricha Shivas, Chief Staff and Strategy, Foundation for Sarcoidosis Research
Elan Shuker, Director Of Development, Programme Partnerships, BBC
Jennifer Shumsky, Founder and Principal, JLS Consulting LLC
Jennifer Siedman, Director of Community Engagement, Courageous Parents Network Inc
Amanda Singleton, Medical Affairs Director, Biopharma & Patient Advocacy, GeneDx
Ted Slocomb, Managing Partner, TS BioAdvisors
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Stephanie Sloves, Senior Director, Brand Strategy, BGB Group
Rachel Smith, Executive Director, Global Head of Rare Disease, CoE, Parexel
Jennifer Snow, Founder and Principal, Apteka LLC
Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics
Mangala Soundar, Senior Director, Discovery Neurology,, PepGen
Steve St. Onge, SVP - Corporate Development, Clarametyx Biosciences
Dionne Stalling, Executive Director, Rare And Black
Stephanie Steele, Director, Immune Deficiency Foundation
Andrew Steinsapir, Acting Chief Technology Officer, Apertura Gene Therapy
Swami Subramanian, Sr. Director, US/Global Pricing, Regeneron Pharmaceuticals Inc
Hiroshi Sugimoto, Director, Takeda Pharmaceuticals
Erin Sullivan, Executive Director, Sisters’ Hope Foundation
Donna Sullivan, Director of patient advocacy, Pathways to Trust
Marshall Summar, Chief Executive Officer, Uncommon Cures
Fabian Suri-Payer, PhD Candidate, Harvard Medical School
Jean Swidler, Executive Director, End the Legacy
Amy Teets, Global Head Patient Strategy, Specialty Care, Sanofi
Pierre A Teulié, CEO, ODDIFACT
Betsy Trainor, Board Member, Ataxia of Charlevoix-Saguenay Foundation
Chelsea Trengrove, CEO and Cofounder, Neoclease
Matt Trudeau, President, ITF Therapeutics
Karen Tubridy, Chief Operating Officer, Abcuro, Inc.
Anna Valsamaki, Senior Vice President of Translation Operations,, BIG Language Solutions
Leon van Wouwe, Clinical Innovation Director, Volv Global
Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio
Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition, BWHI
Colin Werth, Pharma Consultant, Patient Advocate
Melissa Williams, CEO, Simply Patient
Susan Winckler, Chief Executive Officer, Reagan Udall Foundation for the F.D.A.
Jodi Wolff, Founder, Strategic Advocacy Solutions, LLC
Rosangela Wolff Moro, Congresswoman, Federal Chamber of Deputies, São Paulo, Brazil
Sorrel Wolowacz, Head European Health Economics, R.T.I. Health Solutions
Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International
Susan Woolner, Hauenstein Neurosciences Patient & Caregiver Support/Community Partner Manager, Trinity Health MI
Alyssa Wyant, Chief Regulatory & Quality Officer, Praxis Precision Medicines
David Young, President, Research & Development and Founder, Processa Pharmaceuticals, Inc.
Joseph Zabinski, VP, Head of Commercial Strategy & AI, OM1
Samantha Zappia, MS, RAC, Regulatory Affairs Consultant, SK Regulatory Solutions
Sophia Zilber, Vice President, Cure Mito Foundation
Jody Zschiedrich, Head of Strategy, Havas Life Rare
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