2025 Speakers
Mackenzie Abramson, Patient, Advocate, Sr. Manager of Research Program Communications, Global Genes
Anthony Adamkovic, Executive Director, Pharmacovigilance, Harmony Biosciences, LLC
Dr Annette Bakker, President, Childrens Tumor Foundation
Ella Balasa, Patient Engagement Consultant, Patient Advocate
Oliver Barnes, US Pharmaceutical & Biotech Correspondent, Financial Times
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
Thomas Bartlett, Rare Disease Patient Digital Technology Advisor, Bartlett Rare Disease Advocacy
Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research
Lesa Brackbill, Director of Advocacy, Leukodystrophy Newborn Screening Action Network
Christine Brennan, Managing Director, Vertex Ventures HC
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
Raquel Cabo, Head of Patient Advocacy, Communications, Commercial strategy, Ovid Therapeutics
Janet Carlson, CEO, Creative Director, Copy, 1.11 Group
Joel Cartner, Director, Access Policy, Muscular Dystrophy Association
Mrs. Cynthia Cassandro, Vice President, Patient Advoacy, Vigil Neuroscience
James Chambers, Associate Professor, Tufts Medical Center
Jason Colquitt, Chief Executive Officer, Across Healthcare
Colleen Dansereau, Senior Director - Gene Therapy Program and Office of Emerging Medical Discoveries, Boston Children's Hospital
Daniel DeFabio, Rare Dad (Menkes Disease), Co-Founder, Disorder: The Rare Disease Film Festival
Erika Edwards, Health and Medical Reporter and Writer, NBC News
Heidi Edwards, President, Sisters Hope Foundation
Jill Elliott, Vice President, Marketing, X4 Pharmaceuticals
Ms Wendy Erler, Vice President, Global Head Patient Experience & Insights, Alexion – AstraZeneca Rare Disease
Alicia Fiscus, Head of Global Regulatory Affairs and QA, Rhythm Pharmaceuticals
Pangkong Fox, Science Engagement Director, CACNA1A Foundation, Inc.
Albert Freedman, Counseling Psychologist / Rare Dad, Rarecounseling.com
Simon Frost, Chief Executive Officer & Co-Founder, Tiber Capital
Pamela Gavin, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Sonia Gobeil, Cofounder, Ataxia of Charlevoix-Saguenay Foundation
Sonia Gobeil, Cofounder, Ataxia of Charlevoix-Saguenay Foundation
J. Michael Graglia, Co-Founder & Managing Director, Syngap Research Fund
Amy Gray, CEO, Undiagnosed Diseases Network Foundation
James Griffin, Sickle Cell Patient Advocate, Independent Milwaukee Wisconsin
Rachel Harrison, Pre-Approval Access Lead, Argenx
Ms Christina Hartman, Government Affairs, Orchard Therapeutics
Scott Harwood, Director, Check Rare
Guadalupe Hayes Mota, CEO & Co-founder, Stealth Star-up & Senior Lecturer, Massachusetts Institute of Technology
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics
Lauren Holder, Producer/Host, Help 4 HD Live Podcast, Help 4 HD
Patrick Hopkinson, Founder and Managing Director, PHTA Consulting Ltd
Dr Mohammad Imran, Global BD Head, 3H Medi Solution Inc
Dwain Irvin, Chief Executive Officer, NovAccess Global, Inc
Mr. Richie Kahn, Co-Founder & COO, Canary Advisors
Joe Katakowski, Director of Research, RTW Foundation
Jessica Klein, Director, Global Rare Disease Awareness, IPSEN
Connie Lee, President and Chief Executive Officer, Alliance to Cure Cavernous Malformations
Melanie Lendnal, Senior Vice President, Policy & Advocacy, The ALS Association
Veronica Lopez Gousset, MPH, Founder and President, VLG Global
Richa Madan Lomash, Scientific Project Manager, Therapeutic Development Branch, Division of Preclinical Innovation, NCATS, NIH
Dr Peter Marks, Director, CBER, U.S. Food and Drug Administration
Craig Martin, President, Rithm Health; CEO, Orphan Therapeutics Accelerator
Ms Jaclyn Martin, Vice President of Global Head Market Access Cell and Gene Therapy, Bayer
Sweety Mathew, Senior Regulatory Professional, Novo Nordisk
Alice McConnell, Chief Executive Officer, Speragen, Inc
Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research
Ms Jennifer McNary, Co-Founder, One Rare
Ana Mingorance, Chief Development Officer, Loulou Foundation
Mary Morlino, Patient Navigator, Undiagnosed Disease Network Foundation (UDNF)
Ramaiah Muthyala, Professor/Director at University of Minnesota, President and Chief Executive Officer,, Indian organization for Rare diseases
Mr Alexander Natz, Secretary General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Dr Amy Nicole Nayar, Vice President of U.S. Patient Advocacy & Government Affairs, Novartis Gene Therapies
Megan Nolan, CEO, Editor-in-Chief, Rare Parenting
Mr Kevan Oconnor, Venture Investor, Cystic Fibrosis Foundation
Mr Francis Pang, SVP Global Market Access and Geographic Expansion, Orchard Therapeutics
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)
Allison Peck, CFO, Cure VCP Disease
Ms Melissa Penn, Director Patient Engagement Research & Development, Bayer
Mike Porath, Founder And Chief Executive Officer, The mighty
Mr Raymond Puerini, Director, FasterCures, a Center of the Milken Institute
Harsha Rajasimha, Founder and Chairman, IndoUSrare
Harpreet Ram, Principal, EVR Consulting
Rebecca Reimers, Medical Geneticist, Rady Children's Institute for Genomic Medicine
Deborah Requesens, Founder, Society of Hispanic Rare Diseases
Frank Rivera, President, Stronger Than Sarcoidosis
Toni Roberts, Co-Founder, DEBRA South Africa
Christopher Robertson, Professor of Law, Boston University
Mandy Rohrig, Associate Director of Patient Advocacy, BridgeBio Pharma
Luke Rosen, Founder, Rescue 7: Firefighters for Patients
Johanna Rossell, Senior Vice President, General Manager, Sumitomo pharma
Michael Salgaller, Supervisor, Technology Analysis and Marketing Unit (TAMU), National Institute of Health (NIH)
William Sarraille, Adjunct Professor, University of Maryland Francis King Carey School of Law
Ms. Mary Schaheen, President, Prevail Partners Llc
Jeremy Schmahmann, Director, Massachusetts General Hospital Ataxia Center
Carolyn Schwartz, President and Chief Scientist, DeltaQuest Foundation
Jordan Shin, Chief Medical Officer, HAYA Therapeutics
Tricha Shivas, Chief Staff and Strategy, Foundation for Sarcoidosis Research
Jennifer Shumsky, JLS Consulting LLC
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Jean Swidler, Executive Director, End the Legacy
Betsy Trainor, Board Member, Ataxia of Charlevoix-Saguenay Foundation
Daniel Wainstock, Researcher and Rare Disease Advocate, PUC-Rio
Kristin Viswanathan Wolff, Senior Director, Global Government Affairs And Public Policy, bluebird bio
Dr. 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
Sophia Zilber, Board member, patient registry director, Cure Mito Foundation
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