2025 Speakers

I am a dedicated advocate for rare and chronic disease patients, driven by my personal journey and commitment to amplifying the voices of patients, caregivers, and advocacy groups. Diagnosed in my mid-20’s with my first rare disease, I pursued advanced education to bridge the gap between public...

Dean joined the TSC Alliance in November 2018 with more than 17 years of research and development (R&D) leadership in biopharmaceutical and medical device industries, leading teams and technologies from discovery to investigational new drug (IND) and investigational device exemption (IDE), a...

Lynn H. Albizo is the Chief Public Policy Officer for the Immune Deficiency Foundation where she has worked for 8 years. She is an accomplished healthcare policy strategist experienced in state and federal legislative and regulatory advocacy. Her professional experience includes work as a Senior...

Kathryn Alexander is an Equity Capital Markets Consultant advising senior executives of healthcare companies on strategy and investor relations practices.  Kathryn has over 20 years of experience in the financial services industry including a decade covering pharmaceutical stocks and several years...

Amanda Almany is the founder of the Nora Jane Foundation, a nonprofit organization dedicated to advancing research and support for Nonketotic Hyperglycinemia (NKH), a rare and often neglected metabolic disorder. Inspired by her daughter Nora’s diagnosis, Amanda launched the foundation to close...

Ahmad Alrawi is a biopharmaceutical professional with roughly 20 years of experience. He has worked at large-cap pharma (Roche), mid-size biotech (Genentech), and startups (Alexion and RaPharma). Ahmad brings US and global expertise in new product development, commercial operations, business...

Kristin Archibald is the founder and president of NKH Crusaders, a nonprofit organization dedicated to advancing research and support for Nonketotic Hyperglycinemia (NKH). Motivated by her son Thomas's diagnosis with a variant form of NKH, Kristin established NKH Crusaders in 2010 to address...

Barbara is Vice President, and Evidence Generation for Medical Affairs category lead within the Real-World division at IQVIA. Her focus is on supporting the development and operations of real-world evidence research methods for regulators, clinicians, patients and payers. She has worked in late...

Ethan is currently EVP of Business Development at Bionews (www.Bionews.com), where he joined in 2022 following two decades in the health information and community building space.  Bionews provides partnership opportunities to clients through clinical trial recruitment, insights/market research and...

Patient columnist for Rare Disease Advisor, where she shares insights from her personal journey living with cold agglutinin disease (CAD), a rare form of autoimmune hemolytic anemia. Diagnosed in 2020, she uses her voice to raise awareness, educate others, and advocate for improved patient support...

With 34 years of industry experience, Lisa Bair is serial healthcare entrepreneur. She is the founder/ CEO of The Hobart Group (the largest payer agency in the US, which has since been acquired by Precision for Medicine) as well as the founder/CEO of Quantuvis, a SaaS-based solution to support...

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 ...

With a background in biology and firsthand experience as both a patient and a scientist, Ella is deeply committed to amplifying the patient voice in healthcare. Her journey has led her to bridge the gap between research and patient advocacy, particularly focusing on antibiotic-resistant infections...

Matt Baniak’s headshot attached and bio below. I will send Lindsay’s once I have it!   Matt Baniak currently serves as EVERSANA’s Vice President of Client and Delivery Excellence where he is responsible for reimbursement HUBs, digital therapies and complex integrated programs. He also leads...

Caitlyn Barrett, PhD, is a director on the Science Philanthropy Accelerator for Research and Collaboration (SPARC) team at Milken Institute Strategic Philanthropy. Her scientific expertise in cancer biology and neurodegeneration in addition to her experience in grant and program...

Anne Bechet is a bio-pharmaceutical executive with over 20 years of experience in drug development, clinical strategy, general management, and project leadership.  Anne is currently the Global Head of Clinical Development and General Manager for Europe and the USA at JCR Pharmaceuticals, where she...

Natalie Beck, MGC, CGC, is the Senior Manager of Clinical Trials and Real-World Data at Genome Medical. In this role, Natalie leads initiatives to optimize patient engagement in clinical trial opportunities while establishing strong relationships with external partners, healthcare providers, and...

Dr. Berent is a veterinary internal medicine specialist/interventionalist who graduated from Cornell University and completed her residency at the University of Pennsylvania, where she served as an Adjunct Assistant Professor before joining the Animal Medical Center in NYC. She is the Director of...

Lara Bloom is the President and CEO of The Ehlers-Danlos Society and responsible for globally raising awareness of rare, chronic, and invisible diseases, specializing in the Ehlers-Danlos syndromes (EDS), hypermobility spectrum disorders (HSD), and related conditions. Lara leads coordinated medical ...

Dr. Adam Bloomfield oversees the Rare Diseases 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 at AstraZeneca, Sobi, and Moderna. In his current...

Julieta B. Bonvin Sallago is a Lead Clinical Research Associate at the Clinical Trials Division and an Assistant Professor at the Department of Pediatrics. Her background is in pediatric medicine and inborn metabolic diseases. She graduated from the University of Buenos Aires and completed a...

Ian Bonzani is a Senior Principal within the Real-World Technology Solutions team at IQVIA. In this role, Ian leverages his scientific background and RWD consulting experience to design and implement direct-to-patient and decentralized real world research and commercial support platforms across...

Lesa Brackbill is a wife, mother, author, and advocate.  She and her husband Brennan became involved with Krabbe-related advocacy when their daughter Victoria was diagnosed with Krabbe Disease in 2015. Using her knowledge and experience, Lesa helped to reform the Newborn Screening (NBS) program in ...

Teresa A. Brandt, PhD, Vice President, Regulatory Affairs, Avidity Biosciences. Dr. Brandt is a pharmaceutical executive with a strong foundation in molecular biology and drug development. She earned her PhD in Molecular and Cellular Biology from Arizona State University focused on mechanisms of...

Jonathan Bruce is a seasoned biopharmaceutical executive with over two decades of experience in market access, pricing and contracting strategy, and patient services. As Vice President of Market Access, Distribution, HEOR, and Patient Services at F2G Inc., he leads initiatives to ensure patient...

Melody Calkins is the Director of Health Policy at the Biotechnology Innovation Organization (BIO), where she lead’s the organization’s public policy efforts around the implementation of the Inflation Reduction Act (IRA), Medicare Part D, and advocacy on state PDAB regulations. Before joining...

Janet Carlson1.11 Group, CEO/Creative Director, Copy Janet Carlson has always been fascinated by medicine. She toyed with the idea of medical school and opted out twice. Instead, she chose a more creative path that played to her strengths: science, writing, and ideation.  Janet spent eight years...

Joel Cartner is a lawyer and public policy professional working as the Muscular Dystrophy Association’s Director of Access Policy. Cartner leads MDA’s efforts in conceiving, enacting, and supporting the implementation of public policy proposals that expand access to care for the neuromuscular...

Michelle is a Senior Principal with IQVIA Patient Centered Solutions and has more than 20 years of experience with patient-centered research and outcomes measurement, using qualitative, quantitative, and mixed methodologies. Michelle focuses on development and validation of clinical outcome...

Jill Anne Castle, M.Ed has been a state and national advocate in both education and healthcare systems for over 20 years. Jill has designed curriculum and been an adjunct professor of Educational Psychology and Leadership at Mary Lou Fulton Teachers College at ASU. She has been a State Department...

James Chambers 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. As the basis for this research, he developed a novel database of specialty therapy coverage policies issued by...

As a Senior Program Director at Patient Focused Medicines Development (PFMD), I bring over 25 years of expertise in healthcare strategy, patient engagement, and transformative partnerships. Based in Canada, my work bridges the biopharmaceutical, medtech, and non-profit sectors, driving systemic...

Timothy Chen currently serves as a Medical Director of Caliway Biopharmaceuticals. He is a distinguished physician leader with extensive experience in internal medicine and obesity medicine, holding dual board certifications in both specialties. With over 25 years of expertise, Dr. Chen plays a...

R. Duane Clark is General Manager, Rare Diseases, Sanofi.    Duane is a 35+year senior leader in the healthcare industry encompassing Rare Disease, MS, Rheumatology, Transplantation, Oncology and Primary Care therapeutic areas.  Over his career he has held executive leadership positions in...

Jim Coccia is Founder and Partner at TKG Access, a leading consultancy specializing in orphan drug and rare disease access strategies. With over 30 years of experience in the pharmaceutical and healthcare industries, Jim has a proven track record of developing innovative solutions to improve...

With a 24-year career at the intersection of healthcare, life sciences, and public health technology, Jason Colquitt has led transformative initiatives that improve patient outcomes and drive operational efficiencies. As the CEO and Founder of Across Healthcare, Jason spearheaded the development of ...

Dr. Glenna Crooks is a highly respected leader in the field of healthcare policy and rare diseases. She is the Founder and CEO of Strategic Health Policy International, Inc., a  consulting firm specializing in problem solving, strategy and policy development for government and healthcare clients...

Lucy Culp is Vice President, State Government Affairs at The Leukemia & Lymphoma Society (LLS). Lucy leads a team of government affairs staff who advocate for state-level policy change to ensure blood cancer patients have access to affordable, meaningful healthcare. Prior to joining LLS, Lucy...

Ms. Dansereau is an experienced nurse leader with over 40 years of experience in pediatric nursing. Her clinical and operational expertise is in the specialty of  hematology, oncology,  cell and gene therapies. Over the past 20 years, she has focused her career within the clinical research...

Brigid DeCoursey Bondoc is a Partner at Morrison Foerster, a leading global law firm known for its expertise in life sciences. With over 15 years of experience, Brigid specializes in advising pharmaceutical and biotechnology companies on complex regulatory matters related to orphan drugs and rare...

At the age of 12 months Daniel’s first child Lucas was diagnosed with the rare disease Menkes Syndrome. After adjusting his expectations of what raising a child might look like Daniel began to tell Lucas’ story with a short documentary. That film led to Daniel co-founding DISORDER: The Rare Disease ...

Dr. Delatte served as a Senior Pharmacology/Toxicology Reviewer at the US FDA for 12 years. He is an established expert in translating nonclinical data to inform clinical program design, including dose selection, safety monitoring, inclusion/exclusion criteria for clinical protocols, and informed...

My name is Brad Dickerson and I work for a company called iECURE, Inc.  iECURE is a small biotechnology company based in the US that is doing research in metabolic disorders including Urea Cycle Disorders and Phenylketonuria.  iECURE is currently running a global study specifically for newborn boys ...

Elizabeth Donahue is a Senior Director of Project Management in the Peri- and Post-Approval Studies and Real-World Evidence team, PPD, part of Thermo Fisher Scientific. She brings over 18 years of CRO experience to her position. As an operational subject matter expert, Elizabeth is instrumental in...

Sika Dunyoh is currently the Senior Director of Patient Advocacy at Travere Therapeutics. In her role, she develops patient education and engagement strategies for patient communities impacted by rare kidney and metabolic diseases. Prior to Travere, Sika worked at the National Organization for Rare ...

Heidi Edwards is the President and Founder of Sisters’ Hope Foundation. She holds a Bachelor of Arts in psychology from Alvernia University and a Master of Arts in Human Resource Management and Labor Relations from St. Francis University. Heidi works with multiple industry partners, has been named...

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...

Wendy Erler is a seasoned healthcare executive with a passion for improving the lives of patients with rare diseases. As the Vice President and Global Head of Patient Experience & Insights at Alexion – AstraZeneca Rare Disease, she brings a wealth of experience in developing patient-centric...

Eric Feigen is the Regulatory Affairs Manager for the Immune Deficiency Foundation. He leads the organization's regulatory policy efforts to improve access to treatments and encourage the development of new therapies for those affected by primary immunodeficiency. Prior to joining the Immune...

A recognized authority on health plan regulatory and financial issues, Tony Fiori advises health care industry clients on regulations and strategy, and leads policy analyses and projects. Tony works with managed care organizations, hospitals, long-term care organizations, state Medicaid...

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...

Global pharmaceutical pioneer with executive commercial, market access, and government affairs experience across large and small companies with therapeutic expertise in leading organizations specializing in oncology, rare and ultra-rare diseases, and other specialty areas. Vast experience...

Karen has over 30 years of experience in the biopharma industry, in a variety of commercial, medical and strategic consulting roles. She began her career in commercial leadership but moved into the unique area of Expanded Access Programs (EAP) in 2010 when she joined Clinigen, a UK based EAP...

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. As one of the world’s leading therapists specializing exclusively in the rare disease patient...

Dr. Fremeau is an accomplished molecular neuroscientist and biotech entrepreneur with a distinguished record of scientific innovation and leadership at the intersection of target validation, translation, and clinical development. As the Founder and CEO of BrainStorm Therapeutics, he is pioneering...

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 ...

Accomplished biopharmaceutical executive with expertise in R&D, clinical operations, medical affairs, sales, and business development. Led and supported over $1.7 billion in transactions from sourcing to closing. Proven problem solver in CNS, oncology, nucleic acid therapeutics, and gene...

Victoria Gemme is the Director of Policy & Regulatory Affairs at the National Organization for Rare Disorders (NORD). In this role, Victoria is responsible for crafting and implementing NORD’s rare disease policy and advocacy engagement strategy with federal regulatory bodies, specifically the...

Stephen is a pharmacist with over 30 years of experience. He has held several leadership roles within the healthcare industry.. He has a successful track record directed at improving the patient experience and outcomes. He strives for excellence with a strategic focus and strong work ethic build...

Diego is the Executive Director of the Federación Colombiana de Enfermedades Raras (FECOER), an umbrella organisation bringing together 52 patient groups to represent the voice of the rare disease community in Colombia. Diagnosed with pulmonary hypertension over 30 years ago, Diego used his...

Anna Gilmore has been with the FSHD Society since March 2018. In that time, she helped launch several community and patient engagement initiatives, such as the volunteer-driven Chapter program, and the rapidly growing and evolving FSHD Navigator. One of the most dynamic areas Anna is working on...

Christoph Glaetzer, Dipl. Kfm. Chief Global Value & Access Officer Johnson & Johnson Innovative Medicine   Christoph Glaetzer is a leader in advancing the Johnson & Johnson Innovative Medicine global strategy of transformational medical innovation, making accessible new medicines...

Sonia Gobeil is a passionate advocate for Ataxia of Charlevoix-Saguenay (ACS), a rare disease. As the Co-founder of the Ataxia of Charlevoix-Saguenay Foundation based in Montreal (Canada), she has dedicated her career to raising awareness and supporting individuals and families affected by this...

Federico Goodsaid is Senior Vice President for Regulatory and Quality at Ariana Data Intelligence, a US subsidiary of Ariana Pharma in France. At Ariana (since 2019), he leads the alignment of AI product development and regulatory strategies of Ariana Pharma customers. Within Regulatory Pathfinders ...

James Griffin was diagnosed with Sickle Cell at the age of two. He is a Sickle Cell Advocate and a published author who is widely known for his book Breaking Silence: Living With Sickle Cell Anemia. In 2013, he began advocating for sickle cell, and since then he has been using his voice to make and ...

Kurt Gunter, MD, FASCP is a Clinical Development Advisor for Steminent Biotherapeutics and is also Chief Medical Officer at Inozyme Pharma. Kurt has over 30 years of expertise in regulatory affairs and clinical development, focusing on rare diseases and cell and gene therapy. Kurt was previously...

David received his BA from Cornell University & his MA from NYU’s Interactive Telecommunications Program (ITP). David has been on the cutting edge of Internet technology since the early 90s and specializes in designing and building complex digital systems that create efficiencies and better...

Joern-Peter Halle is a seasoned Advisor and Venture Partner at BioGeneration Ventures (BGV), a leading early-stage life sciences venture capital fund based in Europe. With over 25 years of experience in the biotech and pharmaceutical industry, Joern-Peter brings a wealth of knowledge and expertise...

Sonit Handa is an accomplished patient-focused physician executive with wide-ranging experiences across the healthcare value chain - Biopharma, Consulting, Research, Clinical. He has extensive experience in successfully leading cross-functional global teams to launch new companies and...

Christina lives right outside Washington, DC in Bethesda, Maryland with her husband, Matt, and daughters Sophie (13) and Charlotte (8).  Before joining Orchard Therapeutics, she led government affairs for bluebird bio, another rare disease gene therapy company.  She also serves on the board of the...

Guadalupe Hayes-Mota is a healthcare and AI executive, entrepreneur, and policy leader committed to enhancing global medicine accessibility and innovation. He is the founder and CEO of an AI-driven healthcare startup that is transforming drug development and accessibility. Previously, he founded...

Dr Shane Hegarty is Chief Scientific Officer, Co-Founder and IP Co-Inventor at AXONIS Therapeutics, a Boston-based biotechnology company. He has 15+ years of experience leading cutting-edge, award-winning neuro-protection, -modulation and -regeneration research for neurological disorders. In recent ...

Brandon Michael Henry, MD is a physician-scientist and Medical Director of the Cell and Gene Therapy Center of Excellence at IQVIA, where he leads medical strategy for rare disease programs across cell, gene, and RNA therapeutic modalities. He is internationally recognized for his contributions to...

Michele Herndon, DNP, RN, is the Interim Director of Operations for the UDNF. For the past two decades, she has served as a pediatric nurse, leader, and manager in an academic hospital setting. Michele is also the mother to Mitchell who enrolled in the UDN in 2017 after five years of...

MBA Diploma in Health Economics Research University of Keels, United Kingdom BSC Botany University of Sheffiled, United Kingdom Anne Heyes has more than 30 years of experience in indistry and consultancy.  She has expertise in all aspects of market access strategy, evidence development and...

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...

Patrick Hopkinson is a Global Market Access expert with over 25 years’ experience. He is an independent consultant, founder and managing director of PHTA Consulting.  He is a senior leader in the life sciences industry with extensive expertise gained from working across multiple functions in global ...

Wills Hughes-Wilson is Chief Patient Access & Commercial Planning at Mereo Biopharma and a member of the company’s executive leadership team.  She joined Mereo in March 2018 with responsibility for leading the company’s access and commercialisation strategies across the portfolio.   Prior to...

Raymond A. Huml, MS, DVM, RAC, is an executive leader in the healthcare and biopharmaceutical industries, currently serving as the Vice President of Rare & Orphan Disease Strategy at Sciensus, a European life sciences company focused on enhancing patient access, engagement and insights, with a...

Tim joined ARM as CEO in 2022 with more than 20 years of experience in the biotechnology industry. A company-builder with a focus on corporate culture, he has helped early-stage organizations create internal cohesion and advance strategic initiatives. As chief executive, Tim divides his time...

Bilingual Clinical Trial specialist, with more than 10 years of hi-value experience in multi-cultural, multinational Clinical trial, specially for the patient recruitment in Japan and in Asian countries. Accomplished many complex patient recruitment projects as well as business expansion in Asia. I ...

Neil Inhaber, MD, FRCPC Head, Rare Diseases and Marketed Products Medical, Global Medical, Global Portfolio Division, Takeda Pharmaceuticals and Co-Chair, Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease Neil leads portfolio medical affairs strategy development...

Debra Jennings is a transformational Healthcare Leader with 25 years of demonstrated success creating strategies to help elevate culture, patient centricity and business capabilities, predominantly within the pharmaceutical industry. “Deb’s” breadth of experience includes leadership roles in...

Gemma Jennings is Global Vice President of Programme Partnerships at BBC Studios. She leads the organization’s international partnerships strategy and a diverse team of developers, creatives, partnerships experts, producers and marketeers to create meaningful and memorable content campaigns that...

Zhou is an industry veteran with over a decade of experience leading New Product Planning and Commercialization in Nephrology, Hematology, Metabolic, Respiratory, Neuromuscular rare diseases. Currently serving as the Vice President of Early Commercial and Corporate Strategy at Edgewise...

Puja Joshi, MD, is a Medical Director in Clinical Development at Stoke Therapeutics, where she helps develop new treatments for rare genetic diseases. With a background in pediatric neurology and clinical research, she combines her expertise in rare disease drug development with a deep commitment...

Health policy professional by training, clinical researcher by trade, and patient advocate by necessity, Richie is intensely passionate about reducing the time it takes to bring promising, new therapies to the patients that need them most. As Co-Founder & COO of Canary Advisors, he works to...

Dr. Kakkis is Chief Executive Officer, President and a Director of Ultragenyx. He founded Ultragenyx in 2010 to create a company that is uniquely built around a deep and meaningful engagement with patients and their caregivers to fully understand their needs. This includes designing a novel...

Litsa Kapsalis a PhD student in Biological Engineering at MIT, where she develops novel drug delivery systems and formulations, advised by Prof. Bob Langer and Dr. Ana Jaklenec. She is also a Venture Capital Fellow with Portal Innovations, a life science venture investing, creation and development...

A medical doctor, Mwango is an expert in development of analgesic and addiction therapies and post-approval activities (pharmacovigilance, risk management, Phase IV studies). Before joining Parexel in 2018, she spent 16 years with the Food and Drug Administration (FDA), most recently as associate...

Ultra-rare diseases often receive limited attention from the pharmaceutical industry due to the small number of patients affected. The RTW Foundation (RTWF) aims to bridge this gap by funding drug discovery projects focused on ultra-rare diseases, pairing this with free scientific advisory services ...

Louise started her career in clinical research as a study coordinator at the Royal Free Hospital in 2000. Since then, she has worked as a CRA in a biotech and large Pharma in the UK and Australia before joining Fortrea 20 years ago. She spent 10 years in project management, including leading...

Lauren Kopsick is the Founder and Executive Director of The Healthcare Navigation Project (THNP) 501c3, ©2024, Patent Pending. THNP is an interactive method to achieve health equity through grassroots health literacy and life skills training in an immersive environment for anyone 15-115. ...

Lukas Lange is the CEO and Co-Founder of Probably Genetic. Prior to founding Probably Genetic, he worked for the Bill & Melinda Gates Foundation’s $2bn healthcare venture capital arm and for The Boston Consulting Group’s healthcare practice. He is a Rhodes Scholar and holds a PhD in...

Patroski Lawson Founder & CEO A veteran advocate on healthcare and other mission-critical issues, Patroski Lawson has been effectively advising senior executives and leading advocacy efforts in the United States and Europe for more than 20 years. His track record of accomplishment spans both...

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...

Dr. Connie Lee is the founder and CEO of the Alliance to Cure Cavernous Malformation, the patient research foundation representing those affected by cavernous malformation. Dr. Lee is a licensed clinical psychologist and the mother of a daughter with multiple cavernous malformations. Dr. Lee...

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...

Jason Lettiere is Senior Vice President, Corporate & Business Development at Neurvati Neurosciences and GRIN Therapeutics.  He has more than 15 years of experience executing on a range of strategic initiatives including business development and licensing, financial matters, portfolio management ...

Michael Lingzhi Li is an Assistant Professor in the Technology and Operations Management Unit at Harvard Business School. His research sits at the intersection of optimization, machine learning, and healthcare, with a focus on AI-driven decision-making in clinical trials and healthcare...

Naomi has extensive experience working in clinical research and with the rare disease community. She spent many years working at Great Ormond Street Hospital as a clinical research nurse caring for patients and families including those living with rare conditions. As Director, Patient Advocacy at...

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 ...

Veronica Lopez Gousset, MPH has more than 8 years of experience ensuring patients’ voices are represented in healthcare decision-making. Her work includes developing and delivering capability building courses to patient communities on topics such as their role in medicine development and...

Dr. Lu joined the Company in 2020, bringing 15 years of protein therapeutics and small molecule drug development experience including within genetic rare diseases. Previously, Dr. Lu served as the Vice President, Global Program Lead for rare diseases at Takeda (via acquisition of Shire). During...

Hello WODC USA 2025! I’m truly honored to be here at WODC USA! As the Director of Advocacy Relations at Rare Disease Advisor (RDA), I’m excited to connect with so many dedicated voices in the rare disease community. At RDA, our mission goes beyond delivering news—we’re here to empower. Through...

Alastair has 30+ years of experience in the biopharmaceutical development of pre- and post-approval drugs. From 2001-2017, Alastair held a variety of senior positions at AstraZeneca, including laterally as Global Head of Delivery for Real World Evidence (RWE). Here, he had responsibility for the...

Yuri Maricich, M.D., M.B.A., serves as Chief Medical Officer at CAMP4. Dr. Maricich is a “full-stack”, licensed physician-scientist, clinical/therapeutic developer, investor, and strategist. His background includes clinical practice as an internal medicine physician and leading teams and...

Silas Martin is the Head of Access and Policy Research at Johnson & Johnson Innovative Medicine, where he leads a team of researchers focused on generating novel evidence and fostering partnerships in support of policies that enable patient access to transformational innovation.  In his tenure...

Fabiana Marzabal has over 15 years of experience in the pharmaceutical industry supporting pre- and commercial excellence and business insights in rare disease and oncology. Currently serving as the Senior Director of Forecasting, Analytics, and Insights at Viridian Therapeutics, Fabiana is a...

Cara has 20 years of experience working with biotech and life sciences companies spanning corporate, patient, product, and internal communications. Her career has encompassed leading communications and patient advocacy strategies at public life sciences companies as well as advising early-stage to...

Maurizio Mazzi is a dedicated and experienced Senior Associate Director of Patient Advocacy & Professional Relations at Boehringer Ingelheim, a leading pharmaceutical company. With a strong background in healthcare and a passion for making a difference in the lives of patients with rare...

  Reenie McCarthy is the Chief Executive Officer and a member of the Board of Stealth BioTherapeutics.  Reenie also serves on the Board of BIO (Biotechnology Innovation Organization). Reenie is a 20+ year veteran of the investment team of Morningside, Stealth’s principal investor, with extensive...

Katie is the Senior Vice President and Practice Lead of Clinical and Regulatory Services at Lumanity. In this role, Katie drives the strategic direction and operational excellence of the Clinical and Regulatory practice, ensuring alignment with Lumanity’s vision and growth objectives. She leads a...

Juliane K. Mills is a seasoned scientist in the world of clinical research with over 23 years of experience. Juliane's mission is simple but powerful: to make a real difference for people battling rare diseases. At Worldwide, she's the go-to person for creating smart, streamlined plans that ...

Ana Mingorance, PhD, is the Chief Development Officer of the Loulou Foundation, and is focused on accelerating the development of therapeutics for CDKL5 deficiency. Ana is a specialist in genetic epilepsy syndromes and orphan drug development, and is a strong advocate for patient involvement in...

Dr. Maryam Mokhtarzadeh joined Regenxbio as Senior Director for Regulatory Strategy leading global regulatory teams and also heading the Ocular Center of Excellence. Her current roles build on her prior leadership experience at AbbVie as Medical Director of Clinical Development. Before joining...

Juliet Moritz is a dynamic and results-driven leader with over 35 years of experience in the pharmaceutical and biotechnology industry. As the Senior Vice President of Strategic Solutions and Patient Centricity at Ergomed, she is dedicated to driving innovation and improving patient outcomes in the ...

Mary Morlino serves as a Patient Navigator at the Undiagnosed Diseases Network Foundation (UDNF), where she leverages over 17 years of personal and professional experience to address the challenges faced by patients and families navigating the complexities of undiagnosed diseases. Diagnosed with...

Rachit Mukkamala is a senior undergraduate at MIT studying Biological Engineering. His current research in Michael Birnbaum’s lab is focused on the development of a high-throughput platform for TCR sequence assembly. He previously worked in Forest White’s lab, where he used phosphoproteomics and...

Ramaiah Muthyala Received his Ph.D. from the University of Sagar, India; Ph.D. from the University of East Anglia, Norwich, UK; and MBA from the University of St. Thomas, St. Paul, Minnesota. Currently, Ramaiah is an associate professor in the Department of Experimental Clinical Pharmacology at...

Brian joined Saol Therapeutics in 2017 as the Senior Vice President of Commercial Operations.  He brings over 30 years of experience in the pharmaceutical industry and in 2019, assumed additional responsibilities as Senior Vice President, Strategy, overseeing the Commercial and Medical Affairs...

Ramaa Nathan serves as the VP, Data Science and Real World Evidence at EVERSANA with several years of experience in the financial and healthcare industries. Ramaa is a data scientist and statistician with extensive programming, statistical and data analysis skills. She holds a PhD in computer and...

Amy Nicole Nayar, PharmD, MS HEOR is a visionary biotech executive, renowned for her ability to anticipate and navigate the evolving healthcare landscape. She is recognized for pioneering industry firsts in corporate affairs, medical, commercial, and access in the biopharmaceutical...

Katie Niemeyer is a dynamic professional speaker, philanthropist, entrepreneur, and Certified Registered Nurse Anesthetist (CRNA) dedicated to inspiring others to overcome obstacles and achieve their goals. Her remarkable journey began as a teenager when she faced a life-threatening ordeal in a...

Kevan is a life sciences business development and investments professional, and leads infectious disease investments at the CFF. Passionate about partnerships, creativeity, and value creation in the AMR space.  

Eunhee Ohgami is a visionary leader in the field of rare diseases and orphan drugs in pediatric neurology space, currently serving as the CEO of NeuroCores Inc. With a strong background in biotechnology and a passion for improving the lives of patients with rare diseases, Eunhee has dedicated her...

Paula Orandash is a dedicated and passionate Director of Patient Engagement at Ergomed, a leading global clinical research organization specializing in orphan drug development. With over three decades of experience in the pharmaceutical and non-profit healthcare industry, Paula is a seasoned expert ...

Michele M. Oshman is the Chief Patient Advocate and Head of Patient Advocacy Center of Excellence The Biotechnology Innovation Organization (BIO) is the world's largest trade association representing biotechnology companies focused on human health, agriculture, and the environment.  In her...

Dr. Simrit Parmar is founder of Cellenkos Inc., a clinical stage biotech company focused on developing allogeneic T regulatory cell therapy products for treatment of patients with autoimmune diseases and inflammatory disorders. Dr Parmar received her hematology/ oncology fellowship training at...

Dr. Kinnari Patel is President, Head of R&D and Chief Operating Officer for Rocket Pharma, overseeing the global R&D and Technical Operations leadership team with oversight spanning discovery through late-stage development and manufacturing. This includes regulatory, clinical, global...

Allison Peck co-founded Cure VCP Disease, with her husband Nathan who is a patient, in 2018 to drive progress towards treatments for diseases caused by pathogenic variants in the VCP/p97 gene. With backgrounds in industrial engineering and leading companies, they have built a network and the...

Raymond Puerini is a Director for FasterCures, a center of the Milken Institute. He has experience in public health program operations, emergency preparedness and response, and biological sciences within the government, non-profit sector, and pharmaceutical industry. Most recently, Raymond was a...

Donovan Quill is the Executive Vice President of Life Science Strategies at AscellaHealth, a global leader in healthcare and specialty pharmacy solutions serving patients, manufacturers, payers and providers. Overseeing sales planning, new channel development, lead generation, and strategic...

Maha Radhakrishnan, M.D is currently Executive Partner at Sofinnova Investments and in her most recent role was Consultant to the CEO at Dyne Therapeutics helping with the overarching strategy and direction to the company R&D and Commercialization efforts. Prior to that she was the Chief...

After attaining his master’s degree in Health Administration, Patrick has spent his entire professional career working for clinical research organizations within the Early Access and commercial sectors.  During this time, he has held positions within customer service and project management...

Harpreet is the President and founder of EVR Consulting, established in 2011. Harpreet’s pursuits have fervently been directed towards the advancement and execution of programs aimed at facilitating access to vital rare disease medications in low-to-middle-income countries (LMICs). As a visionary...

Dhaval is the Associate Director of Pharmaceutical Sciences at Shionogi Inc based in Florham Park, NJ (subsidiary of Shionogi & Co Ltd, headquartered in Osaka, Japan). He has over 13 years of pharmaceutical drug product development experience and has been instrumental in launch of high impact...

Dorian Readnour is a seasoned professional with over 20 years of experience in the healthcare industry, specializing in patient access across both US and global functions. She began her career after earning a master's degree from the University of Pennsylvania and working at the Children's...

Sabrina is a seasoned expert with over a decade of experience in capturing real-world evidence and patient-reported outcomes. With a strong commitment to amplifying patient voices, she has dedicated the last four years to the rare disease community, focusing particularly on amyloidosis. Sabrina...

Lindsay Reel is an accomplished pharmacy executive with over 20 years of experience in pharmacy leadership, compliance, operations, and business strategy. With a strong focus on driving organizational growth, optimizing pharmacy services, and enhancing both patient and employee experiences, Lindsay ...

Dr Rebecca Reimers is clinically trained in maternal-fetal medicine and clinical genetics. She currently practices pediatric genetics, perinatology, and leads two clinical trials; one in genome sequencing for newborn screening (BeginNGS) and the other in genome sequencing for prenatally diagnosed...

Deborah Requesens, PhD, is the Director of the Orphan Disease Center’s JumpStart Program. This program serves to establish and progress research agendas in emerging and neglected diseases. She partners with patient groups and scientists to encourage scientific collaboration and drive therapeutic...

Dr Sumira Riaz is a Registered Health Psychologist, Cognitive Behavioural Therapist and Founder of Unboxed Psychology, a behaviour change, patient engagement consultancy. Sumira has 18 years of experience working within the NHS, charity section and healthcare agencies specialising in the Patient...

Faisal Riaz is a dedicated and accomplished Medical Director specializing in Neuroimmunology and Alpha-1 Antitrypsin Deficiency (AATD) at Takeda Pharmaceuticals USA Inc. With a strong background in medicine and a passion for rare diseases, Faisal brings extensive experience in the field of orphan...

Luke Rosen founded  KIF1A.ORG  in 2016 following his daughter Susannah’s KIF1A diagnosis. Today, KIF1A.ORG has several programs driving discovery of treatment for neurodegenerative diseases. In November 2022, Susannah was the first child to receive an experimental ASO treatment developed by the...

Johanna has a broad and deep background across consumer goods and healthcare in the U.S. and global geographies. She has led multi-functional teams delivering strong results for companies including Procter & Gamble, Novartis, Merck, Biogen, and Sumitomo Pharma. She has demonstrated success...

Seth Rotberg is a nationally recognized patient leader, healthcare industry professional, and TEDx speaker. After his mother's 17-year battle with Huntington's Disease (HD), Seth made it his mission to improve the patient experience in healthcare. During the past 16 years, he's had a...

As the Head of Global Public Affairs, Rare Diseases at Sanofi, Jean-Pascal leads efforts to evolve the policy environment to ensure that people living with rare diseases have equitable access to diagnosis, innovative treatments, and care. He has nearly two decades of experience in the...

Christian Rubio is Executive Director at EverythingALS, a digital biomarkers and endpoints research non-profit serving the ALS/ MND community through the validation of new measures via remote tracking of disease progression and the lived experience. An experienced advocacy leader who has worked on...

Christopher Rudolf is a seasoned technology entrepreneur and business advisor with over 30 years of experience, specialising in data science, AI, and medical informatics. As the founder and CEO of Volv Global SA, he leads the company's mission to transform healthcare through advanced machine...

Dr. Russo serves as EVERSANA’s Chief Medical Officer.  He is responsible for ensuring the highest levels of quality and data integrity in the design and delivery of projects that require the translation of clinical, population medicine and health economic knowledge into actionable analyses across...

Matt Salo Founder/CEO Salo Health Strategies   Matt Salo is the founding member of Salo Health Strategies, a boutique health care consulting firm in the Washington DC area that specializes in strategic advice, health care policy, Medicaid market development and relationship building across 56...

I am a dynamic and passionate pharmaceutical leader in the patient advocacy space, currently serving as the Global Head of Patient Advocacy at Alfasigma. With over a decade of experience in the pharmaceutical industry, I am dedicated to improving the lives of patients through...

We are the unique investment partner to life sciences companies providing capital to emerge therapeutics and medical devices developing tomorrow's therapies and treatments.

Dr. Carolyn Schwartz is President & Chief Scientist at the not-for-profit DeltaQuest Foundation; and Adjunct Research Professor of Medicine and Orthopaedic Surgery at the Tufts University School of Medicine. A Harvard-trained behavioral scientist, Dr. Schwartz specializes in patient-reported...

As CEO of Legacy Health Strategies, Jessica combines medical and legal expertise with innovative thinking to drive transformative change in healthcare, focusing on integrating patient perspectives to achieve mutually beneficial outcomes. After graduating from Tufts University School of Medicine and ...

Andrew Shao, Ph.D., joined ChromaDex, Inc. in 2019 where he currently serves as Sr VP, Global Regulatory & Scientific Affairs, and is responsible for overseeing the company’s research and development, scientific affairs, regulatory affairs and quality functions. Dr. Shao has spent most of his...

Tricha Shivas joined the Foundation for Sarcoidosis Research (FSR) in December of 2020.  As the Chief of Staff andStrategy, she works with various stakeholders including individuals living with sarcoidosis.  Through her career she has built strong stakeholder relations, created comprehensive...

Elan Shuker is Director of Series Development and Strategy at BBC StoryWorks, where he leads the creative and strategic direction of new programming across genres. With a background in corporate intelligence, storytelling and brand strategy, Elan has worked on high-impact campaigns, combining...

Jennifer is the Director of Community Engagement for Courageous Parents Network (CPN), a non-profit whose mission is to orient and empower parents and others caring for children with serious medical conditions by providing resources and tools that reflect the experience and perspective of families...

Ted Slocomb is a global commercial and market access biopharma executive with deep experience leading the commercialization of high value rare disease, gene therapy and cell therapy products including leadership roles in new product planning, global market access/pricing, HEOR and global marketing...

As Senior Director of Brand Strategy and Behavioral Science, Stephanie leads the development of bold, insight-driven strategies that unlock meaningful differentiation and drive customer behavior change. She partners with brand teams and external stakeholders to shape positioning, messaging, and...

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 ...

Jennifer Snow advises clients on healthcare policy and market trends on the federal and state level. She is a subject matter expert on many topics including the Inflation Reduction Act, the Affordable Care Act, 340B, supply chain dynamics, pharmacy benefit managers and federal health...

Frank Sorgi is President & CEO of FLAG Therapeutics, Inc.  FLAG is developing small molecule therapeutics to treat rare cancers.  FLAG's leading clinical asset is FLAG-003, entering the clinic to treat DIPG (Diffuse Intrinsic Pontine Glioma), a rare and fatal pediatric brain tumor, and...

Steve St. Onge, PharmD, MBA is a biotechnology executive with over 15 years of experience in corporate development, M&A, licensing, medical affairs and clinical practice.  Prior to joining Clarametyx in 2024, Dr. St. Onge led corporate development, licensing and corporate communications at...

Having had a child with a primary immunodeficiency, 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 advocate ...

Dr. Sugimoto is the Director at Takeda Development Center Americas Inc., leading Nonclinical genomic/proteomic biomarker research to support oncology, cell therapy and gastroenterology disease programs. He also serves as the nonclinical pharmacokinetics project representative to support the cell...

Erin Sullivan is the Executive Director of Sisters' Hope Foundation, the global advocacy organization for thosel living with ALSP. She has been an advocate for ALSP since connecting with Sisters’ Hope Foundation when her mother was diagnosed with the disease. Erin is a graduate of Concordia...

    Donna Sullivan is patient advocate, medical educator, writer, film producer, speaker and the proud mom of three kids navigating life with Ehlers Danlos Syndrome. As a trailblazing health advocate, Donna has spent over a decade working to raise awareness of the unique access to care challenges ...

Dr. Marshall L. Summar is a recognized leader in the field of rare diseases, with over 40 years of experience in clinical care, research, and innovation. He is the Chief Executive Officer of Uncommon Cures, LLC, a company specializing in rare disease clinical trials. The company focuses on...

By showcasing untapped medical research to transform off-label usages into approved treatments, Pierre A Teulié is driving a new approach to facilitate the emergence of rare diseases' treatments. His strategic mindset and passion for making a difference have positioned him as a key player in...

Betsy Trainor is an attorney and mother of four residing in Northern Virginia. Her twenty-five-year-old daughter was diagnosed with ARSACS at the age of 20. Betsy now sits on the Board of Directors of the ARSACS Foundation, based in Montreal Canada. She spends a considerable amount of her time...

I build and lead high impact, cross-functional teams to bring innovative therapies to patient communities. I’ve been entrusted over a 30-year career to lead value creation across various therapeutic areas, including oncology, gene therapy, neurology, and rare disease. Along this journey, I’ve been...

Karen Tubridy, PharmD is the Chief Operating Officer of Abcuro, Inc. Abcuro is a clinical stage biotechnology company developing first-in-class immunotherapies for the treatment of autoimmune diseases and cancer through precise modulation of highly cytotoxic T cells. Karen has responsibility for...

As Senior Vice President of Translation Operations at BIG Language Solutions, Anna Valsamaki EMBA leads the development of innovative, insight-driven strategies that drive global growth through mission critical translation in regulated industries, such as Life Sciences, Healthcare and IP. She...

Léon van Wouwe is bringing his clinical development and operational experience to Volv Global SA, to help build bridges between the applied data science solutions the Data Science Team develops and the drug developers, clinical researchers, clinicians, and patients. He has over 20 years of...

Colin Werth is a lifelong resident of Farmville, Virginia and has a degree in IT from Southside Virginia Community College. He works as an IT Specialist for Letterpress Communications. In addition to his work in the IT and marketing field, Colin is a patient advocate for the rare disease...

Melissa is the founder and CEO of Simply Patient where she leads a cross-functional group of consultants passionate about improving patient engagement in biopharma.  She brings nearly 20 years experience in biopharma with a focus on patient engagement in clinical and commercial programs.  ...

Susan C. Winckler is CEO of the Reagan-Udall Foundation for the Food and Drug Administration; the non-profit organization created by Congress to advance the mission of the FDA. Prior to accepting the Foundation post in May of 2020, Winckler served as President of Leavitt Partners Solutions, a...

Sorrel Wolowacz is a seasoned professional in the field of health economics, currently serving as the Head of European Health Economics at R.T.I. Health Solutions. With a strong background in health economics and a passion for improving access to healthcare for rare disease patients, Sorrel brings...

DURHANE WONG-RIEGER, PHD is President & CEO of the Canadian Organization for Rare Disorders, Immediate Past Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency...

Dr. Young has over 40 years of pharmaceutical research, drug development, and corporate experience. He was Founder of Processa Pharmaceuticals, Inc. and served as CEO from 2017-2023 before becoming President of R&D. Dr. Young was a Board Member and Chief Scientific Officer at Questcor...

Joseph Zabinski, PhD, MEM is VP, Head of Commercial Strategy & AI at OM1. He oversees design, implementation, and evolution of OM1's commercial strategy, including prioritization, go-to-market planning, and alignment with emergent market needs. Dr. Zabinski also serves as subject matter...

Sophia leverages her professional background and personal story to create meaningful contributions to the rare disease community. She has over 20 years of experience with drug development, including clinical data analysis, and managing statistical programming teams to ensure successful delivery of...

Jody Zschiedrich is a seasoned rare disease commercial strategy expert with over 20 years in the pharmaceutical and biotech industry. Across his career, he has designed and orchestrated more than 30 disease state education and brand launch campaigns. Jody holds a BS in Engineering and an MBA in...