2026 Speakers

I am first and foremost a wife to the love of my life and a mama to two incredible teenage boys. I am a sister, a daughter, an aunt, and a friend. Those roles are the most important parts of who I am — and how I want to be remembered.   I am also living with hereditary ATTR amyloidosis, the same...

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 ...

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 ...

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...

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 ...

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...

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...

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 ...

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...

Grant has nearly two decades of commercial, business development, finance, and operations experience in the biopharma and medical device industry. Prior to joining the Sentynl team in 2015, Grant led marketing operations for Amedra Pharmaceuticals and Lineage Therapeutics, which was successfully...

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...

Donna Cowan is a Certified Clinical Research Coordinator and Project Manager with more than 20 years of biopharmaceutical experience.  For the past several years Donna has concentrated on the management of Investigator Initiated Studies and Expanded Access Programs (Compassionate Use, Managed...

Axel de Tonnac is a Clinical Solutions Partner at Rivia, supporting biotech companies in strengthening clinical trial data visibility and decision-making. Prior to joining Rivia, he worked at SOPHiA GENETICS, focusing on data-driven solutions in precision medicine. He holds a Master’s degree in...

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...

Kelly Esperias, EdD, MBA, CFRE, serves as Chief Strategy & Operations Officer at the National Organization for Rare Disorders (NORD®), the leading and longest-standing patient advocacy group for the 30 million Americans living with a rare disease.  An accomplished and dynamic executive who has...

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...

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...

Al Freedman, PhD, is a psychologist, educator, and rare disease advocate with more than two decades of experience working with individuals and families affected by rare diseases and disabilities.  Dr. Al is inspired by his personal journey as the father of Jack, who lived with spinal muscular...

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...

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...

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...

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...

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...

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...

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...

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...

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 ...

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...

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...

Joan Kincaid is the Founder and CEO of And Scene, a patient experience insights platform for rare disease pharma teams. She has spent 25 years building SaaS platforms at the intersection of technology and human connection — from enterprise consulting to community engagement platforms. The last...

Lauren Kopsick is a healthcare navigation educator and systems strategist working at the intersection of healthcare, education, and workforce development. She is the Founder and Executive Director of The Healthcare Navigation Project, where she designs active-learning programs that strengthen...

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...

Cornelia Lee, PsyD, is the founding Chief Executive Officer of the Alliance to Cure Cavernous Malformation (formerly Angioma Alliance), the leading international patient advocacy organization for individuals and families affected by cerebral cavernous malformation (CCM), a rare neurovascular...

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...

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 ...

Craig is a deeply experienced executive, advisor and board director, with extensive background in biotech and health technology fields. His focus in emerging innovation and desire to make an impact have led to a longstanding commitment to supporting rare disease communities. Craig has led...

Marc co-founded Minoryx in 2011 and has been its CEO since its inception leading its journey from concept to its current stage. Its lead drug candidate, leriglitazone, is now under review by the EMA for a potential marketing authorization in X-ALD, while in the US is in phase 3. Minoryx has...

Maurizio Mazzi is a dedicated and experienced Senior Associate Director in Patient Advocacy & Professional Relations at Böhringer Ingelheim. With a strong background in healthcare and a passion for making a difference in the lives of patients with rare diseases, Maurizio has been instrumental...

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...

Over the past 20+ years, Amanda Moore has had the pleasure of gaining depth and breadth of experience in multiple roles across clinical development operations, program leadership, and research.  She has led cross-functional programs and contributed to development activities of four approved and...

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...

Kris O'Brien has nearly four decades of experience in the clinical research industry, spanning roles in contract research organizations (CROs), pharmaceutical companies, and academia. Throughout her career, she has worked across a wide range of therapeutic areas, with a strong focus on rare...

Paula Orandash is a dedicated and passionate Director of Patient Engagement at Ergomed. With over three decades of experience in the pharmaceutical and non-profit healthcare industry, Paula is a seasoned expert in patient-centric approaches to drug development and rare disease advocacy.Paula’s...

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 ...

Luis A. Rojas, PhD, is a Senior Research Scientist Statistician and executive leader in clinical development, regulatory-aligned evidence generation, and natural history data strategy. He serves as CEO and President of InCSD LLC and Vice President at Indication BioScience, with prior executive...

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. Robert Ryan is a visionary leader in the field of rare diseases and orphan drugs,  and currently serves as the CEO of Paradigm Therapeutics, where he spearheads the development of an innovative therapy for Epidermolysis Bullosa (EB).  With a career spanning over 35 years in the pharmaceutical...

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 ...

Jessica Sheldon, MPH, serves as a Feasibility Strategy Associate Director at Parexel International LLC, where she focuses on feasibility for rare and genetic disease clinical development. Driven by a deep commitment to improving outcomes for individuals living with rare and genetic conditions—as...

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...

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 ...

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...

Frank L. Sorgi, Ph.D., MBA, is President, CEO and founder of FLAG Therapeutics. He brings with him 30 years of experience as an entrepreneurial scientist in the field of drug delivery and dosage form design and as a business executive in the biotechnology and pharmaceutical industries. Prior to...

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...

Laura Tadvalkar is a Managing Director on the Venture Team at RA Capital Management. Laura works on new company creation and early-stage investments, and serves as a Board Director for Hemab, Hyku Biosciences, and Typewriter Therapeutics. She previously served as a Board Director for Mariana...

Hillary Valley, PhD is a Mission Ventures Investments Principal at the Cystic Fibrosis Foundation (CFF), where she works closely with industry partners to advance transformative therapies for cystic fibrosis. Hillary earned her B.S. in Chemistry and Biology (double major) from St. Lawrence...

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...

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 ...

Susan C. Winckler, RPh, Esq., is CEO of the Reagan-Udall Foundation for the Food and Drug Administration. The Foundation is the non-profit organization created by Congress to advance the mission of the FDA. Prior to accepting the Foundation post, Ms. Winckler served as President of Leavitt...

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 ...