Learn from world KOLs in rare diseases on the latest in AI & digital health, gene therapy, clinical trials, patient access and more. Ensure that you are equipped with the most recent policy and governmental changes in OMP legislation and HTA.
With a footfall of 2,000+ attendees, exhibitors will get to speak directly to their target market, expand sales leads, strengthen and establish their brand and close deals with customers on-site.
Get access to our dedicated event app (5 weeks before the event), see all of our 2,000+ attendees and set up 1-2-1 meetings* to maximise your ROI in attending the congress.
*A ticket with full app access is required to use all messaging and meeting features
Meet start-ups or pitch your company at the leading rare disease congress. Meet new companies that can help your business or investors that can take you to the next level.
Host or participate in our pre-congress workshops where we deep dive into topics including AI, digital health, patient data, global access & pricing, next gen therapies, and global joint clinical assessment and more with all key stakeholders.
These sessions are intimate discussions and one of the best ways to find out what your target audience wants.
The media zone will be bustling with all of the biggest names in the rare disease world. Witness live interviews and use our zone to announce press releases and social media outreach to maximise your presence.
World Orphan Drug Congress USA is proud to announce its recent accreditation by The CPD Group, a globally recognised accreditation body for quality professional development. This accreditation signifies World Orphan Drug Congress USA commitment to delivering high-quality educational experiences that contribute to the professional growth of its attendees.
Join 2,000 attendees at the world's largest orphan drug and rare disease event.
This is your best opportunity in 2026 to network with professionals in the rare disease industry.
Lynn H. Albizo is the Chief Public Policy Officer for the Immune Deficiency Foundation, where she has worked for 9 years. She is an accomplished healthcare policy strategist experienced in legislative and regulatory advocacy. Her previous professional background includes serving as a Senior Analyst ...
ANNETTE BAKKER, Ph.D., CEO — CHILDREN’S TUMOR FOUNDATION (CTF), Chair (ad interim) of CTF Europe Annette Bakker, a Ph.D. in Biochemistry, was an academic researcher for ten years - University Antwerp, Yale Medical School, and the Myology Institute Paris. Following this, she accumulated 15 years of ...
Chris Garabedian founded Xontogeny in June of 2016 to support multiple promising technologies from early development through clinical proof of concept. In 2017, Chris joined Perceptive Advisors to develop their Venture Fund strategy and is Portfolio Manager of the Perceptive Xontogeny Ventures Fund ...
Kimberly LeBlanc is a genetic counselor and the Director of the Undiagnosed Diseases Network (UDN) Data Management Coordinating Center in the Department of Biomedical Informatics at Harvard Medical School. The UDN Data Management Coordinating Center oversees the activities of the UDN, a network of...
Rob, a former All-American punter at Syracuse. In 2010, during his senior season, he was diagnosed with a rare form of brain cancer. His treatment lasted 16 months, ending his NFL aspirations. A Syracuse University graduate, Rob earned a B.S. from the Whitman School of Management and a Master’s in ...
With a first degree in Biochemistry and Pharmacology, and a Ph.D. focusing on drug metabolism and Pharmacokinetics, Graeme has worked in the areas of Drug Safety and Medical services for over 35 years. Having worked as a Head of Global Pharmacovigilance for a multi-national innovator Company and EU ...
Dr. Bedrosian joined Amylyx as Chief Medical Officer (CMO) in 2023, bringing to the company nearly 30 years of experience in building successful clinical development and translation research programs in the pharmaceutical industry. Dr. Bedrosian served most of her nearly six years at Ultragenyx as...
Michael was first introduced to rare diseases while a student in college when his father was diagnosed with a rare form of cancer. As with many rare families, this experience launched him on an odyssey for answers. He began his career with Senator Orrin Hatch, Republican of Utah where he developed...
Sarah Glass, PhD, is Chief Operating Officer at the n-Lorem Foundation, where she leads global operations to bring individualized RNA-based medicines to patients with nano-rare diseases—those affecting only a single individual worldwide. Trained as a geneticist and driven by her personal experience ...
Will Greene is a rare disease researcher and patient advocate based in the San Francisco Bay Area. He serves on the board of the Foundation for Prader-Willi Research (FPWR), where he provides strategic oversight and supports efforts to accelerate research, advocacy, and community engagement. He is...
Elise Hoover joined the Foundation for Sarcoidosis Research in May of 2024. She is a strategic leader with a decade of experience in rare disease research initiatives and national collaborative partnerships. As VP of Research, she convenes clinicians, researchers, patients and their families, and...
Luke Rosen is a community leader who works urgently to bring treatment and care to children living with neurological diseases and cancer. Luke is an advocate for firefighters and families affected by 9/11 related cancer, and is a cancer survivor himself. He is the CEO of US Special Rescue, founder...
Dr. Neva West has 20 years of pharmaceutical industry experience, including as a Global Medical Director at Biogen and as the owner of a medical communications and strategy firm working with many mid-size and startup pharmaceutical companies. She has worked in many therapeutic areas, spanning from...
Caroline Allen is a dedicated and passionate leader in the field of rare diseases and orphan drugs. With over a decade of experience in patient advocacy, she currently serves as the Head of Patient Advocacy at ITF Therapeutics. Caroline is committed to improving the lives of individuals affected by ...
Karen Kahn is the Co-Founder of the ForeBatten Foundation and the mother of twin daughters, Amelia and Makenzie, who were diagnosed with juvenile Batten disease (CLN3) in 2017, just shy of their seventh birthday. Karen earned her Bachelor of Architecture degree from the University of Virginia and...
James Griffin was born in Milwaukee, Wisconsin and diagnosed with sickle cell disease at the age of two. Drawing on his personal journey as a patient, James brings a unique and deeply informed perspective to his advocacy work. He began championing sickle cell awareness in 2013 and has since become...
Joe Katakowski is the Director of Research at the RTW Foundation (RTWF), where he leads initiatives that empower rare disease parents and patient advocacy groups as central drivers of therapeutic development. He believes that success in ultra-rare diseases is far more likely when efforts are led by ...
Christina lives right outside Washington, DC in Bethesda, Maryland with her husband, Matt, and daughters Sophie (13) and Charlotte (8). Christina leads government affairs for Orchard Therapeutics, a Kyowa Kirin company and gene therapy leader focused on ending the devastation caused by genetic and...
Sophia Zilber is an advocate, data expert, and rare disease leader who blends deep personal commitment with over two decades of professional experience in drug development. With a background in clinical data analysis and statistical programming, she brings both expertise and heart to everything she ...
Daria Julkowska, PhD, is a European research strategist and rare disease leader recognized for building some of the world’s most ambitious collaborative frameworks in rare disease research and innovation. Trained as a molecular biologist in France and Poland, she transitioned from bench science to...
Julia is a senior associate at Atlas Venture where she focuses on investing in and building companies that develop novel therapies to improve patients’ lives. Julia serves as a board observer for nChroma Bio, Pheon Therapeutics, Renasant Bio, and a stealth newco. She was previously a board observer ...
Boris Sevarika is a pharmacist and nanomedicine scientist driven by the question of how biological therapies can be delivered more effectively to patients. Originally from Belgrade, Serbia, he completed his secondary education at the Third Belgrade Grammar School before moving to Germany in 2016 to ...
Rachel has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development ...
Dr. Adam Bloomfield is Global Head, Medical Affairs and oversees the General Medicine, Rare Disease and Pediatrics Medical Affairs team at Premier Research. He brings to Premier extensive US and global experience in medical affairs leadership and clinical development strategy from his previous work ...
Ben Wilson is a dedicated advocate for rare diseases, particularly Bloom Syndrome, as a parent of a child affected by this condition. With a deep personal understanding of the challenges faced by families dealing with rare diseases, Ben has become a prominent voice in the rare disease community. As ...
Deborah Ebert Long, MD is Senior Vice President of Medical Affairs at CSL. In this role, Deborah leads CSL’s worldwide Medical Affairs function, partnering across regions and functions to advance medical strategy, gather scientific insights, and engage healthcare professionals and key stakeholders...
Sarah White, MPH, has served as Executive Director of the MRCT Center since 2018, leading strategy, operations, and financial stewardship to advance the Center’s mission and expand its impact across the clinical research community. With more than 25 years of experience across large academic medical ...
Marybeth McAfee, MA, GC, is Vice President of Community and Medical Affairs at the National Organization for Rare Disorders (NORD®), where she leads Medical Affairs, Community Engagement, Membership, Educational Initiatives, and Information Services. She also oversees NORD’s Rare Disease Centers of ...
Maryann Kirby is a dedicated professional in the field of human biospecimens as the Director of Business Development at Donor X, a innovative organization that is changing the way we think and secure human biospecimens for research, process development and medical device testing. Maryann has spent ...
Sandra Talbird designs and programs decision-analytic economic models, including cost-effectiveness, cost-utility, cost-benefit, and budget-impact models using Excel and Visual Basic for Applications. She has also developed mathematical models including compartmental dynamic transmission models for ...








































































































