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...

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...

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...

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...

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...

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...

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...

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...

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...

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 ...

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

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 ...

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...

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...

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...

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...

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...

Davide Garrisi holds a doctorate degree in pharmaceutical science and technologies by the Univesrity of Padua (Italy) and an executive MBA by ALTIS/Catholic Univesirty of Milan. Davide started his career in the API manufacturing industry and since 2005 he worked in the CRO Industry, where he...

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...

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 ...

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...

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 ...

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...

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 ...

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...

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 ...

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. ...

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...

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...

Richa Madan Lomash is a Scientific Project Manager in the Therapeutic Development Branch (TDB) within NCATS where she manages rare disease late-stage preclinical drug development projects across different modalities and therapeutic areas. Since joining NCATS in 2020, she is an integral part of the...

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...

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...

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...

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...

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.  

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...

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...

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...

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...

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...

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...

Dr. Michael Salgaller leads the Technology Analysis and Marketing Unit (TAMU) within the NIH’s Technology Transfer Office, where he leverages over 20 years of business, scientific, and investment experience in various life science sectors to support technology development and commercialization. The ...

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...

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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...

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...

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...

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...

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...

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...

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...

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...