Why Attend - America's Largest Rare Disease Event

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 ...

James D Chambers, PhD, is an investigator at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and a Professor of Medicine at Tufts University School of Medicine. He developed the SPEC Database, a novel resource of specialty drug coverage policies issued by major...

Dr. Djordje Filipovic.is currently serving as the CEO of AB2Bio, a Swiss Biotech Company focused on developing best in class innovative therapies for the treatment of Interleukin-18 (IL-18) driven systemic inflammatory disorders with high unmet medical needs. Djordje’s background includes more...

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 ...

Federico Goodsaid is a regulatory strategy consultant at Regulatory Pathfinders and Ariana Data Intelligence. He was CEO of MBQ-Pharma, a biotech company licensing IP developed at the University of Puerto Rico. The goal of MBQ Pharma is to commercialize anticancer drugs developed through this...

Debra Jennings is a dynamic and transformational healthcare leader with over 25 years of proven success driving strategies that elevate organizational culture, strengthen patient-centric models, and enhance business capabilities across the pharmaceutical industry. Her extensive expertise spans...

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 ...

Dr. Devyn Smith brings significant cell and gene therapy development and platform expertise from his 20+ year career. Devyn joined Arbor Biotechnologies as Chief Executive Officer on April 27, 2021 after concluding his role as Chief Operating Officer of Sigilon Therapeutics. Prior to...

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 ...

Charlene Son Rigby is the Chief Executive Officer of Global Genes. She has spent her career building organizations at the intersection of data, technology, and life sciences. Charlene was previously Chief Business Officer at Fabric Genomics and held executive roles at enterprise software and...

Jessica Cerullo is a dedicated and passionate Senior Director of Patient Advocacy & Disease States Initiatives at Pierre Fabre Pharmaceuticals. With over a decade of experience in the pharmaceutical industry, Jessica has established herself as a leader in advocating for patients with rare...

Amanda Forys is Managing Partner at Magnolia Market Access. Her consulting and manufacturer experience has covered a wide range of commercialization topics, including health policy, pricing and rebating, coding, coverage and reimbursement, health economics, budget impact modeling, patient...

I am the Founder & CEO of Biovance Consulting LLC, and a physician-executive with a unique blend of medical, commercial, and operational leadership. I specialize in helping high-growth biopharma companies focused on bringing Rare Disease companies bring life-altering therapies to market with...

Dr. Paul August has dedicated his career to transforming scientific discovery into tangible hope for patients battling rare and neurological diseases.  Dr. August currently serves as Chief Scientific Officer at ReviR Therapeutics, where he spearheads innovative research programs for devastating...

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...

Salman Bhai, MD is a neuromuscular neurologist and biotech executive specializing in clinical development and translational strategy for rare diseases. He serves as Vice President of Clinical Development at Secretome Therapeutics, where he leads clinical and scientific efforts supporting the...

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...

Dr. Kakkis is best known for his work developing novel treatments for rare diseases and working on policy issues affecting rare and ultra-rare disease treatment.  He is Chief Executive Officer, President and a Director of Ultragenyx. He founded Ultragenyx in 2010 to create a company that is...

Dr. Michelle McClure is a geneticist and the Director of Public Policy for the American College of Medical Genetics and Genomics (ACMG). Her professional interests include improving policies related to rare diseases, ranging from genetic testing and diagnosis to development and coverage of gene...

Having had a child with primary immunodeficiency (PI), I invested in the medical profession and trained to become a medical assistant at the Ohio Institute of Health Careers. My focus has always been on helping patients and guiding them to needed resources. I spent a decade in the PI consumer...

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...

Yael Weiss Is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical...

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...

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...

Alex Meixner is the Vice President of State Policy at The ALS Association, leading a team of government relations and grassroots professionals as we pursue our mission to make ALS livable and cure it. Prior to joining The ALS Association in 2022, he worked for over a decade at the American Heart...

Katie McCarthy is a seasoned professional with over 25 years of experience in the biopharmaceutical industry. As the Senior Vice President and Practice Lead of Clinical & Regulatory at Lumanity, Inc., she plays a pivotal role in driving the company's strategic initiatives and client...

Caitlyn Barrett manages SPARC’s work in the Rare Diseases and Cancer focus areas. Barrett supports philanthropists, family offices, and non-profit organizations to develop funding strategies that support biomedical research. She also serves on the Board of Directors of the Coalition Against...

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 ...

Mary joined the Foundation for Sarcoidosis Research as the organization's first-ever President and Chief Executive Officer in 2020. As President and CEO, Mary serves as the primary representative and spokesperson for FSR and leads the organization's strategic vision with patient...

Tricha Shivas joined the Foundation for Sarcoidosis Research (FSR) in December of 2020.  As the Chief of Staff and Strategy, she works with various stakeholders including individuals living with sarcoidosis and their loved ones, academic researchers, clinicians, industry partners, regulators and...

I proudly earned my medical degree from the Universidad de Buenos Aires, which set the foundation for my journey in pediatrics. My residency at Hospital Interzonal Agudos Especializado en Pediatría Sor María Ludovica in La Plata, Argentina, deepened my commitment to caring for children in...

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...

Guadalupe Hayes-Mota is the Director of the Bioethics Program at Santa Clara University's Markkula Center for Applied Ethics Santa Clara University and a Senior Lecturer at MIT. Born with severe hemophilia in rural Mexico, his personal experience with rare disease drives his commitment to...

A veteran leader in the rare disease patient focused drug development movement, Annie joined the EveryLife Foundation in 2018, where she’s led numerous, community-driven evidence development efforts including the National Economic Burden of Rare Disease study, The Guide to Patient Involvement in...

Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law...

Christine Green is the Co-Founder and President of Finding Hope for FRRS1L, where she is a leading advocate for parent-led innovation in rare disease drug development. Her work focuses on redefining traditional therapeutic development models by positioning families and patient organizations as...

Melanie Lendnal is the Senior Vice President of Policy & Advocacy at the ALS Association, where she leads the organization’s public policy efforts to make ALS a livable disease while working to find a cure. The organization works at all levels of government and in all branches of government in...

Liz is President and a co-founder of The MYT1L Project Foundation. The MYT1L Project is a parent-led, scientist-supported foundation established in 2025 to advance research into evidence-based interventions for MYT1L Neurodevelopmental Syndrome, a rare genetic condition marked by developmental...

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 ...

Lara Bloom is President and CEO of The Ehlers-Danlos Society, where she leads global efforts to raise awareness of rare, chronic, and invisible conditions—specializing in Ehlers-Danlos syndromes (EDS), hypermobility spectrum disorders (HSD), and related conditions. She spearheads international...

Jo Kaur is a civil rights attorney turned drug developer. Jo’s story is a mother’s fierce fight to prolong her son Riaan’s life and to build treatments for Cockayne syndrome, a fatal genetic disorder that causes profound neuronal loss and early childhood death. Riaan was diagnosed with Cockayne...