2026 Speakers
June 9 - 11, 2026
Boston Convention & Exhibition Center
Mackenzie Abramson, MPH, BCPA, Patient Advocate
Rebecca Ahrens-Nicklas, MD, PhD, Assistant Professor of Pediatrics Director, Gene Therapy for Inherited Metabolic Disorders Frontier Program, Children's Hospital of Philadelphia
Lynn Albizo, Chief Public Policy Officer, Immune Deficiency Foundation
Caroline Allen, Head of Patient Advocacy, ITF Therapeutics
Paul August, Chief Scientific Officer, ReviR Therapeutics
Annette Bakker, CEO, Childrens Tumor Foundation
Anny Bedard, President, Egetis Therapeutics US
Camille Bedrosian, Chief Medical Officer, Amylyx Pharmaceuticals
Hourinaz Behesti, Founder & CEO, Hebbian Bio
Salman Bhai, Vice President, Clinical Development, Secretome Therapeutics
P.J. Brooks, Deputy Director, Division of Rare Diseases Research Innovation, Member of IRDiRC Funders Constituent Committee (FCC), NIH/NCATS
Jonathan Brudvig, Senior Director, Scientific Evidence, Amicus Therapeutics
Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience
Jessica Cerullo, Senior Director, Patient Advocacy & Disease States Initiatives, Pierre Fabre Pharmaceuticals
James Chambers, Professor, Tufts Medical Center
Jason Colquitt, Chief Executive Officer, Across Healthcare
Anne Corvelle, President, Board of Directors, FD/MAS Alliance
Eric Crombez, MD, EVP, Chief Medical Officer, Ultragenyx
Rumi Desai, Director, Global Regulatory Strategy, Amgen, Inc.
Michael Eging, Executive Director, Rare Access Action Project
Lisa Emrick, Medical Director, Neurogenetics, Baylor College of Medicine
Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics
Eric Feigen, Regulatory Affairs Manager, Immune Deficiency Foundation
Djordje Filipovic, Chief Executive Officer, AB2 Bio Ltd.
Amanda Forys, Managing Partner, Magnolia Market Access
Karen Frascello, Senior Director, Medical Affairs, Alnylam Pharmaceuticals
Albert Freedman, Counseling Psychologist / Rare Dad, Rarecounseling.com
Chris Garabedian, Portfolio Manager, Venture, Perceptive Advisors
Sarah Glass, Chief Operating Officer, n-Lorem Foundation
Federico Goodsaid, SVP Regulatory, Ariana Pharma
Will Greene, Board Member, Foundation for Prader-Willi Research
Sonit Handa, Member, Board of Directors, The Josselyn Center
Sonit Handa, CEO & Founder, Biovance Consulting
Rachel Harrison, Director, Managed Access Program, ARGENX
Alex Hersham, Co-Founder, Nonsense Tx
Elise Hoover, Vice President of Research, Foundation for Sarcoidosis Research
Neil Inhaber, Head, Marketed Products, Global Medical, Takeda
Debra Jennings, Head of Patient Services Operations, North America, Kyowa Kirin
Richie Kahn, Board Member, One Rare
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Karen Khan, Co-founder, ForeBatten Foundation
Paul Kidwell, Podcast Host / Producer & Columnist, In Sickness, Men and the Caregiving Culture
Bonnie Kiesling, Head of US Marketing, ITF Therapeutics
Ned Kitfield, CCO, Ambit RD, Inc
Jonathan Kornstein, VP, Rare Disease & Pediatrics Clinical Development, Clinipace Inc. d/b/a Caidya
Graeme Ladds, CEO, PharSafer
Patroski Lawson, Co-founder, The Just Society
Kimberly LeBlanc, Director, UDN Data Management Coordinating Center, Harvard Medical School, Harvard Medical School
Julianna LeMieux, Deputy Editor in Chief, Genetic Engineering & Biotechnology News
Robert Long, Executive Director, Uplifting Athletes
Peng Lu, Chief Medical Officer, Pharvaris
Fabiana Marzabal, Sr Director Forecasting, Analytics and Insights, Viridian Therapeutics
Maurizio Mazzi, Senior Associate Director, Patient Advocacy & Professional Relations, Böhringer Ingelheim
Katie McCarthy, SVP & Practice Lead, Clinical & Regulatory, Lumanity, Inc.
Michelle McClure, Director of Public Policy, American College of Medical Genetics and Genomics
Jennifer McNary, Co-Founder, Canary Advisors
Alex Mexiner, Vice President of State Policy and Advocacy, The ALS Association
Kiran Musunuru, M.D., Ph.D., Barry J. Gertz Professor for Translational Research, Perelman School of Medicine University of Pennsylvania
Brian Nappi, SVP, Strategy, Saol Therapeutics
James Oakley, CEO, Keep Me Breathing
Francis Pang, SVP Global Market Access and International Geographic Expansion, Orchard Therapeutics
Dr. Rupesh Patel, Director, Global Patient Safety Quality Management, Exelixis
Dave Pearce, Chair, International Rare Diseases Research Consortium (IRDiRC)
Matthew Prentice, Director of State Policy, Immune Deficiency Foundation
Dorian Readnour, Vice President, International, Sarepta Therapeutics
Stephanie Rider, Director, Payor Relations & Policy, Immune Deficiency Foundation
Frank Rivera, President, Stronger Than Sarcoidosis
Luis Rojas, Chief Clinical Development Officer, Polaryx Therapeutics
Luke Rosen, Founder, Rescue 7: Firefighters for Patients
Mrs Jennifer Shumsky, Payer Relations, Little Hercules Foundation
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Devyn Smith, CEO, Arbor Biotechnologies
Charlene Son Rigby, Chief Executive Officer, Global Genes
Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics
Steve St. Onge, SVP - Corporate Development, Clarametyx Biosciences
Dionne Stalling, Executive Director, Rare And Black
Ying Tam, Chief Scientific Officer, Acuitas Therapeutics
Chelsea Trengrove, CEO and Cofounder, Neoclease
Fyodor Urnov, Scientific Director, Innovative Genomics Institute, Professor, Department of Molecular & Cell Biology, University of California at Berkeley
Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio
Yael Weiss, Chief Executive Officer, Mahzi Therapeutics
Neva West, CEO & Co-founder, Biotech Social, Inc
Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International
Timothy Yu, Attending Physician, Investigator, Division Of Genetics And Genomics, Boston Children's Hospital
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