2026 Speakers

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 ...

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...

Caitlyn Barrett manages SPARC’s work in the Rare Diseases and Cancer focus areas. She also serves on the Board of Directors of the Coalition Against Childhood Cancer. Previously, Barrett was the senior director of research and programs at CureSearch for Children’s Cancer. She also established the...

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...

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...

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...

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...

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...

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 ...

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 ...

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...

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...

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...

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...

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...

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...

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...

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 ...

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...

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 ...

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 ...

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...

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...

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...

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...

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...

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...

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...

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...

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...

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...