2024 Speakers

Samuel Agyei Wiafe is a clinical psychologist who, after running into a family with undiagnosed syndrome, immediately had clear what the impact of these diseases can be on the people affected and those close to them, as well as the incredible challenge they pose for the entire health system. And...

Based in New York and operating from the US office, Dennis holds an MSc in Finance, has over 20 years of BioPharma industry experience, and is a recogized thought leader in pre-approval access and with specific expertise in clinical trials. Over 8 years at myTomorrows, his role is pivotal in...

Derek Ansel is Executive Director, Therapeutic Strategy Lead, Rare Disease at Worldwide Clinical Trials.  Mr. Ansel has worked in clinical research for more than a decade with a focus almost exclusively in rare and pediatric diseases, including non-malignant hematology, autoimmune...

Kevin Bagley, DHA, MBA, FACHE is passionate about finding ways to improve the systems of care that support Medicaid participants across the country. Kevin’s most recent role was as the Medicaid Director for the state of Nebraska, serving under two Governors, Pete Ricketts and Jim Pillen.  Prior to...

Mark has more than 25 years of experience managing client relationships across a number of pharmaceutical and biotech service providers, and has spent the past decade focused on the early and expanded.access landscape.

Kevin Baruzzi is a partner in Simon-Kucher’s Healthcare and Life Sciences division based in the Boston office. He primarily supports pharmaceutical and biotechnology clients. He is a credentialed veteran with over a decade of progressive growth in the life sciences strategy consulting arena. He...

I am responsible for Inceptua's Early Access business unit, supporting pharmaceutical and biotech companies in making their medicines available to patients in need before commercialization through Expanded Access, Named Patient, Compassionate Use and Early Access mechanisms.  

Dr Benesh is a neuroscientist and molecular biologist by training with over 15 years of experience in basic and clinical research. She currently serves as a Director of Therapeutic Expertise in ICON's Centre for Rare Diseases and focuses on strategy and design of rare disease clinical...

Kara Berasi started working in the patient advocacy space after her son was diagnosed with a rare disease called PMM2-CDG, or congenital disorder of glycosylation. She has a personal passion to change the landscape of care for those living with rare diseases, and brings that tenacity and focus to...

Alexandre Bétourné, Ph.D., Pharm.D., is Executive Director for the Rare Disease Cures Accelerator-Data and Analytics Platform initiative and previously served as its Scientific Director. Dr. Bétourné works with the RDCA-DAP team to expand its reach into new diseases areas accessing new data and...

As Co-founder and Chief Executive Officer of Nanoscope Therapeutics, Sulagna Bhattacharya leads a talented and diverse team dedicated to the development of sight-restoring therapies for the millions of patients worldwide blinded by inherited retinal diseases for which no cure exists. ...

Robin is a Director of Biostatistics at Veristat.  She oversees the operations of the biostatistics department and collaborates with all the integrated operational teams throughout the clinical trial  and regulatory submission process to help produce high quality statistical deliverables. She has...

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...

Morgan Boname, M.S., is Senior Director of Public Policy at Neurocrine Biosciences, where she leads the Public Policy team and is responsible for a portfolio issues that promote the development and delivery of medicines that aim to relieve suffering for people with great needs in...

Natasha Bonhomme brings nearly two decades of nonprofit and maternal and child healthexperience to her role as Founder of Expecting Health at Genetic Alliance. She launchedExpecting Health to bring together a range of patient/family and professional partners to addressthe need for...

James Boomhower, MS(c), FP-C, NR-P, CCISM has been involved in EMS for over 20 years in a variety of health systems.  He currently functions in the role of Critical Care Transport Specialist-Paramedic/ Lead Peer Support Director with Boston Medflight of Bedford, Massachusetts His desire to bring...

Wendy Borsari graduated in 1990 with a degree in Psychology from Mount Holyoke College. She joined Tenaya Therapeutics in July of 2021 to create their patient advocacy department Prior to this she worked as a consultant with both large and small pharmaceutical companies helping them to incorporate ...

Julie Breneiser is the Executive Director of the Gorlin Syndrome Alliance (GSA). Prior to her position as director, Julie served as volunteer Board President for the GSA. She and her two young adult children are affected with Gorlin syndrome, a rare genetic disorder caused by a tumor suppressant...

Staff Profile: Philip John (P.J.) Brooks (nih.gov)  

Bert Bruce is the US President, Rare Disease at Pfizer, Inc. where he is responsible for commercialization of a portfolio of medicines for Rare Cardiology, Hemophilia, Sickle Cell Disease, Endocrine and Metabolic diseases, and preparation for a future potential portfolio of medicines, including the ...

Chuck Bucklar   Chuck has over 30 years of commercial and general management experience in the biotechnology/pharmaceutical industry. During that time, he has held senior positions with increasing responsibilities at companies including BioMarin, GPC Biotech, and Cephalon. He has also served as a ...

Karin Butler has a BSc (Hons) in Neuroscience from the University of Bristol, an MSc in Health Economics and Decision Modelling from the University of Sheffield and is the Head of Technical Research at YHEC.  Karin has been the technical economic lead on submissions to multiple agencies...

Dr. Robert M. Califf was confirmed as the 25th Commissioner of Food and Drugs. As Commissioner, Dr. Califf oversees the full breadth of the FDA portfolio and execution of the Federal Food, Drug, and Cosmetic Act and other applicable laws. This includes assuring the safety, effectiveness, and...

As Senior Evidence Lead at HealthLumen, Josh manages the epidemiological, health policy and health economics research that informs HealthLumen’s modelling approaches, which focus on gaining unparalleled insights into the current and future burden of rare genetic diseases and other non-communicable...

I coordinate the creation of RARE Revolution Magazine’s digital content, liaising with stakeholders from across the rare disease ecosystem. Our website—and our core magazine and spotlight editions—offers a platform for people living with a rare disease (PLWRD) to share their stories. It is a...

Cynthia Cassandro, BS, RN Vice President of Patient Advocacy, Vigil Neuroscience Cynthia Cassandro is a biotechnology leader and dedicated advocate for patients living with rare diseases. With over 20 years in industry and 10 years working in patient access and advocacy, Cynthia has extensive...

James Chambers is an investigator at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, and an Associate Professor of Medicine at Tufts University School of Medicine. His research has highlighted variation in patients’ access to specialty and orphan medications and...

Chère Chapman completed her biology honours degree from Dalhousie University (King’s College) and set out for a year inrural Japan as part of a global teaching and cross-cultural exchange program of the Japanese government. Aftertwo years back doing her master’s at University of Toronto, Chère...

Ellen Cho is the Director of Specialty Programs at Direct Relief, a health-focused humanitarian aid organization based in Santa Barbara, CA. In her role, she works with pharmaceutical manufacturers and healthcare providers to provide longterm access to lifesaving therapies for people living with...

Priya has a PhD in Biochemistry and currently VP and Head of Clinical Bioanalytics & Translational Sciences at Beam Therapeutics. She manages a function within clinical that works across R&D and holds responsibility for bioanalytical, biomarker and forward & reverse translational...

R. Duane Clark is General Manager, Rare Disease, Sanofi. Duane is a 30+year leader in the healthcare industry with a professional and vocal passion for the rare disease community. Over his career he has held executive leadership positions in General Management, Sales, Marketing, Commercial...

Kyle has over 15 years’ experience in the life sciences industry with expertise in commercial and market access strategies, access planning, and value communication. At Red Nucleus, he has also been busy assessing value, setting price, and developing innovative contracts, with specific focus on...

Jason Colquitt is a seasoned technology executive and rare disease patient with a fervent dedication to advancing innovation and transformation within the healthcare and clinical research sectors. With 25 years of experience, Jason has emerged as a trailblazer in pioneering disruptive technologies...

Matt has spent over 10 years with leading roles in the field of mobile technology with specializations in mobile healthcare technology. In 2017 he earned a Global MBA from Southern New Hampshire University. In 2019 he co-founded a nonprofit digital media organization focused on advocacy for...

Ron Cooper served as President and Chief Executive Officer of Albireo Pharma and a member of its board of directors from 2015-2023, prior to the acquisition by Ipsen Pharma for over $1Billion of value. Under Mr. Cooper’s leadership, Albireo successfully became a public company focused on the...

Donna Cowan is a Certified Clinical Research Coordinator and Clinical Project Manager with more than 15 years of experience in the biopharmaceutical field coordinating and managing Phase 2, 3, 3B and 4 (post marketing) trials, Investigator Sponsored Trials (IST) and Collaborative Research (CR)...

Carole Cramer is President and CoFounder of BioStrategies, a preclinical biotech start-up focused on delivering biologics to hard-to-treat tissues and CNS. BioStratgies has a proprietary delivery platform exploiting natural plant lectins (sugar-binding proteins) to direct the uptake and...

Andrew has more than three decades of experience in senior international commercial leadership positions within the Pharmaceuticals and Medical Devices sectors. With a specialization in early access pre-approval unlicensed medicines, he holds key responsibilities in leadership, commercial...

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...

Michelle joined the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) as Executive Director in February 2016. The majority of her career has been spent working in the nonprofit sector; seven years of which were spent leading a nonprofit chamber of commerce training and...

Recognized leader in clinical research living at the intersection between cultural innovation and emerging technologies. As a creative builder and change agent, her mission is to promote a human centered healthcare system built on empathy and radical collaboration: where patients are integral...

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 ...

Thomas DeFay, PhD, Deputy Head of Diagnostics Strategy and Development at Alexion AstraZeneca Rare Disease At Alexion, Tom serves as Deputy Head of Diagnostic Strategy and Development, overseeing diagnostics and screening approaches such as Artificial Intelligence, Genetics, and Newborn...

Linda Dell is a seasoned pharmaceutical executive with over 25 years of program and project management experience in the life sciences space.  In her role at ReAlta she shepherds their development programs from preclinical stage through clinical development and ultimately to the threshold of...

Jin Ding is a Research Fellow at iHuman since 2021, collaborating with Professor Paul Martin and Professor Michael Hopkins on a Wellcome Trust funded project. Her primary focus lies in understanding the industry dynamics and evolution of orphan drugs across the UK, EU, and US regions. Leveraging...

Lauren is the Chief Operating Officer at TREND Community. Lauren has deep experience scaling rapidly growing businesses in the U.S. and abroad. She played a pivotal role in the expansion of C Space from a startup to a global customer agency and most recently served as Chief Operating Officer for...

Leslie was diagnosed with a rare neuroendocrine pituitary tumor that was causing Cushing’s Disease in 2012.  After two unsuccessful surgeries, removal of her pituitary gland, meningitis, blood clots, recurrence, radiation, and a decade of blood draws, procedures, and medications, she declared...

Michael Eging is the Founder and Executive Director of the Rare Access Action Project (RAAP), a non-profit based out of Washington, DC, focused on navigating patient access to rare disease therapies. Michael began his journey as an advocate when his father was diagnosed with a rare cancer. At that...

Sean is founder and CEO of Collaborations Pharmaceuticals, Inc. which is focused on developing and using machine learning and AI approaches for rare and neglected disease drug discovery. Sean graduated from the University of Aberdeen; receiving his M.Sc., Ph.D. in Clinical Pharmacology and D.Sc. in ...

Shaun is the Vice President, Global Market Access for Specialty Ophthalmology at Johnson & Johnson Innovative Medicine.  In this role he is responsible for shaping the global development and execution of payer value dossiers across the therapeutic area. In his 18 years in the pharmaceutical...

Erik A. Feingold is the CEO and Co-founder of FDNA Inc. He previously served as the Senior Advisor to the CEO, and as President & Head of Strategy at Sharecare (NASDAQ: SHCR). He joined Sharecare following the acquisition of his company, FeingoldTech GmbH. Feingold is a distinguished business...

James is a neuroscientist with >10 years of experience in studying rare neurodevelopmental disorders. His background and expertise include electrophysiology, synaptic function and plasticity, and stem cell-derived neuron differentiation. James received his Ph.D. from the University of...

Alicia is the Head of Global Regulatory Affairs at Rhythm Pharmaceuticals with over 20 years of industry experience. Joining Rhythm in January 2020, she helped systematically build the regulatory and GCP/GLP quality assurance functions in support of multiple programs and molecules and played a key...

Pawel Fludzinski became CEO of AmideBio in May 2018. Prior to AmideBio, he had a 31-year career with Eli Lilly and Company, with 23 years in executive leadership positions in the United States, United Kingdom, and Japan, encompassing roles in strategy, research and drug development. In addition, he ...

Ben has worked in the field of biomedical research since 2009. His experience ranges from nearly a decade working at the bench in basic science, to five years working in the clinical research space. Currently, Ben is responsible for translational research projects at Sanford Research. His team...

Lindy has been working in the biotech, pharmaceutical and medical device industries for the past 24 years with a focus on strategic development of submission dossiers and finding novel solutions for access issues. Lindy’s background in prior pharmaceutical positions in clinical research and health...

As the Head of Global Public Affairs, Rare Diseases and Rare Blood Disorders 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 two decades of experience in ...

(7) Sebastian Franzinger | LinkedIn

Mike Fusakio has more than five years of experience in Regulatory Affairs and Project Management in both contract research organization, academic, and consulting settings. He has experience with regulatory strategy and project management for initial INDs and NDA submissions, ex-US projects (MHRA...

Renata Gallagher, MD, PhD, Medical Director at Parexel has 20+ years of clinical experience in the care of adults and children with rare inborn errors of metabolism.  She is the former Director, Biochemical Genetic Medicine, University of California, San Francisco and was the Principal Investigator ...

With a rich background in patient advocacy and a keen focus on rare disease, Neha brings over 20 years of dedicated experience to the table. Through her various positions, most recently as the Global Head of Patient Advocacy and Engagement at Ascendis Pharma, she has been instrumental in driving...

Dr. Sarah Glass is the Chief Operating Officer of n-Lorem Foundation. Sarah received her Ph.D. in Molecular Genetics at Ohio State University where she trained in rare inherited cancer syndromes. She has over 20 years of experience in clinical development and research across...

Leslie Gordon is the mother of a child with Progeria, co-founder of The Progeria Research Foundation (PRF), and serves as PRF’s volunteer Medical Director. Dr. Gordon is the Principal Investigator for ongoing Progeria programs, including the PRF International Progeria Registry, Medical and Research ...

Deb is the SVP, Development Operations with over 25 years of experience in industry. She has spent the last 12 years focused on rare and ultra-rare genetic diseases applying her experience in drug development to help bring treatments from preclinical stage through to the clinic and beyond.

Heidi Grabenstatter, Ph.D., is the Scientific Director of RDCA-DAP.  She has previously served as the Science Director of the International Foundation for CDKL5 Research (IFCR) managing the research portfolio for the organization.  Heidi worked closely with IFCR’s Board of Directors, MSAB, Centers...

Alicia Granados, MD & PhD in Public Health from the University of Barcelona. She is a certified specialist in respiratory medicine. Currently serving as Global Head Rare Disorder Medical Scientific Advocacy & Insights at Sanofi, where she has been heading its global Health Technology...

Nathan Grant is the twin brother of Nik Grant who has mucopolysaccharidosis (MPS) type II, also known as Hunter syndrome. Inspired by Nik, Nathan has engaged in numerous research and advocacy initiatives to help people with rare diseases and their families. Currently, Nathan is a Principal...

Amy Gray is the Chief Executive Officer of the Undiagnosed Diseases Network Foundation (UDNF). Amy leads the UDNF with more than two decades of extensive fundraising, operations, and patient advocacy experience shaped by her previous roles working with four national non-profit voluntary health...

Brandi Greenberg services as the VP of Marketing for Real Chemistry's Analytics & Insights group. She joined that team seven months ago after spending several years leading marketing and customer insight for Optum Life Sciences. She transitioned into that role after a nearly 20 year career...

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 an...

Gay has been advocating in the rare disease community for over twenty-five years advocating for genetic testing, shared data, and patients leveraging their data. Gay is an expert on private insurance and social services through state and federal policy for those affected by various disabilities.   ...

Director of Client Services at Splash Clinical LLC Responsible for developing, nurturing, and managing relationships with clients to ensure their satisfaction with the services provided, drive awareness of Splash Clinical's solutions and collaborate with cross-functional team to ensure...

Matthew Hanchard, PhD is a Research Fellow working in academia, with two books, fifteen articles, eight years of teaching acumen in higher education, and extensive industry experience. He works around pharma, data, and open science. At present his work focusses on health economics arguments over...

Costello Medical provides scientific and health economic support in the analysis and interpretation of clinical and economic data. My team focusses on supporting biopharma companies in overcoming the unique challenges in rare diseases, often navigating very limited evidence bases. Our services...

Sue is on a mission to help organizations do change better. Known for keen insights, memorable imagery, and a system-level perspective, she empowers leaders and teams to drives lasting change. She combines research-based approaches from disciplines including change management, team...

Dr Shane Hegarty is Chief Scientific Officer, Founder and Inventor at AXONIS Therapeutics, Boston-based biotechnology company. 15+ years of experience leading cutting-edge, award-winning neuro-protection, -modulation and -regeneration research for neurological disorders. As Research Fellow in...

Alexandra Heumber Perry has been appointed Chief Executive Officer of Rare Diseases International (RDI ) in June 2023. She brings 20 years’ experience in global health with demonstrated capabilities in patient advocacy and multi-stakeholder partnership, focusing initially on neglected and...

Gary Ho, co-founder of the Gout Support Group of America and Senior Community Director at TREND Community, is a dedicated advocate for rare diseases and underserved communities. His leadership and empathy have effectively driven awareness and support initiatives, contributing to better healthcare...

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...

Huong Huynh is a pharmacologist with over 20 years combined experience working in academia, non-profit organizations, and regulatory agency. She is the Director of Regulatory Science at Critical Path Institute (C-Path) and Professor of Practice at The University of Arizona James E. Rogers College...

Oxana Iliach, PhD Sr. Director, Regulatory Strategy Dr. Iliach has more than 15 years of experience in the healthcare industry including the last 10+ years in regulatory affairs.  She has expertise in developing and executing regulatory strategies for drugs for rare diseases including...

Dr. Intson is a neuropharmacologist specializing in rare neurological disorders. Her venture-backed startup, Varient, crowdsources the most effective off-label therapies for rare and genetic disease symptoms. This highly-scalable method creates touch points with, and collects natural history data...

Sun-Gou and his team uses large datasets and computational tools to support decision making across early discovery research and late stage clinical programs at BridgeBio. This includes identifying and validating targets by quantifying human genetic support through the use of diverse population...

Gretchen Jordan is a dedicated advocate for pituitary health. After a successful career as a global supply chain software professional, she made a life-changing decision to prioritize personal health and quit her job to undergo treatment for Cushing's Disease. The experience ignited her passion ...

Andria Joseph has a BSc (Hons) in Biomedical Sciences, an MSc in Neuroscience from King’s College London and a Executive MSc in Health Economics, Policy and Management at the London School of Economics and Political Science (LSE). Andria is a Project Director (Outcomes Research) at YHEC and has...

Chris Juday applies over 25 years of experience in life sciences to connect with clients on a range of issues centered on health care quality, value, and access. Having worked in a variety of life sciences roles, Chris can translate access and value concepts into practical solutions for Real...

Richie is a public health professional by training, clinical rresearcher by trade, and patient advocate by necessity. Over the last 14 years, he has worked across the clinical trials industry to reduce the time it takes to bring promising new products to the patients that need them most. As...

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...

Principal (Partner) at IQVIA, leading Information management, Analytics consulting practice for IQVIA, incuding next Gen insight and GenAI solutions for life sciences. In his role, he is responsible for conceptualization, and oversight of solutions for the life sciences clients with focus on...

Experienced biotech/pharma entrepreneur with more than 25 years of experience in developing and marketing a diverse therapeutic area products.   Dr. Katakam founded three companies, raised equity and debt capital, and managed operations leading to successful high-valued corporate exit strategies.   ...

A veteran leader in the rare disease patient focused drug development movement, Annie joined the EveryLife Foundation in 2018, where she’s led the Cost of Delayed Diagnosis: A Health Economic Study, the National Economic Burden of Rare Disease study, the development of the ICD Code Roadmap, and the ...

ADRIAN KIELHORN, DIPL.-OEK. Accomplished Health Economist and Market Access Specialist Adrian has spent most of his career working for pharmaceutical companies in health economics and outcomes research (HEOR) roles. He broadened his experience working in market access, strategic...

Maria Kirsch is a decisive, strategic and forward-thinking leader with more than 20 years of experience across operational excellence and strategic development in the pharmaceutical industry. She previously served as Head of Patient Experience, Technology & Innovation at Novartis, where she...

Akihiro Ko has served as Chief Executive Officer and Chair of the board of directors since May 2017, when he co-founded Elixirgen Therapeutics with Minoru Ko. Previously, Mr. Ko served as Chief Operating Officer for Elixirgen, LLC, the parent company of Elixirgen Therapeutics, and President of...

With over 20 years in healthcare technology, Bret Koncak's expertise was honed during his tenure as an executive at Cerner (now Oracle Cerner), a renowned healthcare IT organization. His role frequently placed him in the midst of healthcare practitioners from esteemed doctors to dedicated...

Lauren Kopsick, caregiver, has an eclectic background, that includes a VP in banking, corporate marketing training and development, small business consulting, call center management, volunteering and board memberships. She also cofounded the Richmond, Virginia, mixed pediatric/adult Support Group...

Ivan leads Real Chemistry’s Real-World Data Solutions team (IPM.ai). The solutions team designs methodologies to uncover and understand patient and HCP communities in the US. He has supported the commercialization and clinical development of 80+ disease-modifying therapies for patients suffering...

Rahul is a Project Director at Allucent, specializing in cell and gene therapy studies. He currently leads the Cell and Gene Therapy Initiative within Allucent, providing comprehensive training and support to team members working on cell and gene studies. Rahul has over 22 years of experience in...

Neha Kumar currently leads Global Commercial Strategy for Precision Medicine at Johnson & Johnson (J&J) Innovative Medicine, focusing primarily on precision medicine for rare diseases. She is a passionate advocate for the transformative power of genomics in rare disease diagnosis and...

Sheryl (Sherry) Lard, PhD, is the Associate Director for Product Management (ADPM) in the Center for Biologics Evaluation and Research (CBER) at the FDA. Dr. Lard began her tenure in CBER as the Center’s Associate Director for Quality Assurance, leading the center’s product jurisdiction and ombuds...

Dr. Connie Lee has been the CEO of the Alliance to Cure Cavernous Malformation (formerly Angioma Alliance) since its founding in 2002.  The Alliance to Cure is the only patient research foundation for cerebral cavernous malformation in the US and has led the way in multiple critical areas: the...

Experienced medical affairs professional with a demonstrated history of working in the pharmaceutical industry. Entrepreneurial mindset with the ability to excel in an autonomous operating environment. Experienced at developing strategic medical plans across multiple regions/ countries. Expertise...

Andrew W. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT's Computer Science and Artificial Intelligence Laboratory. His healthcare-related research interests...

Veronica Lopez Gousset has over 8 years of experience driving international multi-stakeholders initiatives toward patient-centered healthcare change.  Veronica’s work spans into the field of rare diseases working closely with patient communities in Europe, South America and Africa to elevate the...

Cat Lutz, Ph.D., M.B.A. is the Vice President of the Rare Disease Translational Center at The Jackson Laboratory (JAX). With 25 years of experience in mouse genetics, Dr. Lutz has focused her research efforts on patient organizations and families diagnosed with rare diseases. The JAX Rare Disease...

Gary Hutchinson is SVP and Managing Director of Modality Solutions, a biopharmaceutical cold chain engineering firm specializing in global cold chain validation. For the last twenty years, he has designed life sciences supply chain logistics and biopharmaceutical cold chains. He is a speaker for...

Craig Martin is a CEO, founder, and consulting executive with 25+ years’ experience working with biotech, pharma, health tech, and healthcare organizations. He specializes in helping companies developing transformative technologies to build awareness, value and relationships critical to advancement ...

Jackie is currently leading the Global Market Access Team for Cell and Gene Therapy at Bayer.  She is responsible for the market access strategy, design, and capability build to secure favorable market and patient access across the globe for Bayer’s entrée into cell & gene therapy.  Bayer’s CGT ...

Sweety Mathew is a Global Regulatory Affairs professional and a Global CMC Product Lead (Rare Diseases) working with Novo Nordisk. She has 10+ years of experience and has a broad expertise across biopharmaceutical drug development (Novels, peptides (synthetic & recombinant) and...

Annette Maughan is a seasoned CEO, entrepreneur, and brand builder with a history of establishing and leading a range of mission-driven organizations.    Earlier in her career, Annette established Digital Aces, an international neutral host operator and digital infrastructure company.   In...

My professional interests have always been driven by my deep desire to improve communities and solve problems. I have years of experience administering both private and government contracts, designing, planning, coordinating, scheduling, estimating, and managing large complex multi-year engineering ...

Father/Founder searching for a path to an approved therapy for his daughter and all of the children affected by LBSL.

Kristin is a well-known and lifelong member of the Hunter syndrome community. Her late brother, Zachary, gave her the drive to spread awareness about MPS II from a young age. The diagnosis of her son, Charlie, just three years after the passing of her brother kickstarted her passion for fighting...

Stuart Mealing is an Associate Director of York Health Economics Consortium and leads the Consortium’s health economic modelling and patient reported outcomes workstream.  He is a member of NICE's Highly Specialised Technologies Evaluation Committee and the NIHR Invention for Innovation (I4I)...

Dr. Laurence Mignon brings more than 20 years of experience to n-Lorem as an seasoned academic researcher and clinical developer with a passion for rare diseases. Most recently, Laurence worked at Ionis Pharmaceuticals both in the Translational Medicine group and the Neurology Franchise Clinical...

Juliane K. Mills is a seasoned explorer in the world of clinical research with over 20 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...

Samar is an inventor & serial entrepreneur with 20+ years experience in Biomedical Sciences. He is Co-Founder of several Biotechs and Biomedical device /diagnostic companies (developed & commercialized capital biomedical instruments). Samar has published more than 200 papers and held...

James F. Mongiardo has extensive experience in building companies. He seves as Chief Executeive Officer of A-Synaptic Corp. Previously, he has served as Chief Executive Officer for public biotechnology and healthcare services companies and as head of United States Marketing for Schering-Plough...

Mary Morlino is a Patient Navigator at the Undiagnosed Diseases Network Foundation (UDNF). She has over 16 years of personal and professional experience understanding the challenges patients and families face during an undiagnosed disease journey. Diagnosed with Sarcoidosis, a multi-system rare...

Lindsay Cardinal Murphy is a seasoned voice of Integrated Intelligence as a Senior Group Director at Real Chemistry, where she empowers healthcare, life sciences, and pharmaceutical clients through data-driven research. With over 15 years of experience, Lindsay has honed her expertise in...

Prof. Ramaiah Muthyala is an associate professor in the Department of Experimental Clinical Pharmacology, an Adjunct Professor in the Department of Medicine, Graduate Faculty, Bioinformatics, and Computational Biology, formerly Senior Assistant Director, Center for Drug Design, and associate...

Ramaa Nathan is a Director of Data Science 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 information science from ...

Dr Nizar has 20 years of rare disease patient advocacy strategy experience focused on development of nano-rare therapies and patient-centered outcomes. She has deep expertise in diversity, equity, and inclusion (DEI) strategy and in Middle Eastern/Asian patient advocacy landscapes with experience...

Mr. Palko is a strategic Pharmaceutical Industry commercial executive with significant leadership demonstrated in US and Global Marketing, US Sales, Franchise and Portfolio Strategy, New Product Planning and Business Development and Advertising Agency Client Management. Currently, Greg is VP, North ...

Jim Palma is CEO of TargetCancer Foundation. Since joining TargetCancer Foundation in 2010, Jim Palma has overseen its growth from a small start-up to a nationally recognized foundation supporting comprehensive rare cancer research programs and patient support services. Prior to joining...

Effie Parks, originally from beautiful Montana, has become a guiding light in the rare disease community following her son Ford's diagnosis with CTNNB1 syndrome. Settling in Washington, she transformed her family's journey into a crusade for advocacy, support, and empowerment for families...

David Pearce is President of Innovation & Research for Sanford Health.  He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986.  He gained his PhD in 1990 at the University of Bath, UK.Dr. Pearce has been researching...

Sue Peschin, MHS, is president and CEO of the Alliance for Aging Research, the leading nonprofit organization dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health. The Alliance’s vision is to create a ...

Peter Pitts is President of the Center for Medicine in the Public Interest, a Visiting Professor at the University of Paris Medical School, a Visiting Scholar at the New York University School of Medicine, Division of Medical Ethics, and a Research Fellow at the US-Israel Education Association. He...

Otto Prohaska received his Ph.D. in Applied Physics at the University of Technology in Vienna, Austria, for the development of multiple microsensors for brain research. Rotary presented him with the Leonardo da Vinci Prize for his outstanding work in this field. Dr. Prohaska came to the U.S. as...

After graduating from ESCP Europe, a Paris Business School, in 1996, Xénia Proton de la Chapelle worked in leading positions in various big international companies (Ernst & Young, Elis Group, Generali Group) before running her own textile company for 8 years. After the birth of her 4th kid...

Harsha K Rajasimha, Ph.D., is a serial entrepreneur, investor, and philanthropist. Harsha is Founder and CEO of Jeeva Clinical Trials Inc.(https://jeevatrials.com) a AI-driven unified platform for sponsors to execute modern clinical trials with efficiency and universal accessibility. Harsha serves...

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...

 Joshua Resnikoff is the co-founder and CEO of TMA Precision Health, a company at the forefront of utilizing whole genome sequencing in precision medicine guidance for rare disease patients. An entrepreneur and biomedical engineer, Resnikoff has a rich background in both consumer and biotech...

Dr. Ritchie is the CEO and Chief Scientific Officer of Med-Life Discoveries, a Canadian biopharma company developing plasmalogen-based therapeutics for RCDP and other neurological conditions. He obtained his PhD in Biochemistry from the University of Saskatchewan and has accrued over 20 years of...

Frank Rivera, a rare disease patient advocate, an Illumina Ambassador, A RDLA PatientAdvisory Council Member, Patients Rising Senate Member, a pharmaceutical advisory member andambassador, a speaker for the rare disease community as well as a patient of Sarcoidosis. I also haveSjogren's...

Christopher Robertson is N. Neal Pike Scholar and Professor of Law at Boston University.  Robertson served as the reporter for the Health Law Monitoring Committee of the Uniform Law Commission, and is an elected member of the American Law Institute Robertson's more than 50 scholarly articles...

Devin Rosenthal, Vice President & Head of Due Diligence at NovaQuest Capital Management, has broad operational and strategic experience across a range of therapeutic areas, stages of development, and company types within the biopharmaceutical industry. As a member of the NovaQuest Product...

Heidi Ross is the Acting Vice President of Policy and Regulatory Affairs at the National Organization for Rare Disorders (NORD). Heidi has built a career out of her passion for domestic and global health policy, and she joined NORD in January 2020. Prior to joining NORD, Heidi worked for nearly...

Yury Rozenman is the Senior Vice President of Business Development at Ada Health. In this role, he leads the company's strategy, vision and oversees execution of all global business development activities in life sciences/pharmaceutical area. Yury has considerable experience of working in and...

Christopher is the CEO and chairman at Volv Global, an applied data science company. Data Science, AI, Machine Learning and Data are the lifeblood of Volv Global SA, and with Christopher's extensive experience and vision in medical informatics, his aim is to bring precision and evidenced-based...

After leaving the Mayo Clinic, from 1988 to 2007, he held academic, clinical and administrative leadership positions as Chief of Cardiac Surgery, Pediatric Cardiac Surgery and Heart Transplantation  at several Academic Hospitals in the USA, including Temple University, St Christopher’s...

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

Over the past decades, Mr. Salvatierra has built effective relations between the public and private sectors in the healthcare industry. He has been successful in planning and directing all aspects of the organization's strategies, objectives and initiatives. Prior to founding the Americas...

Rachael Sawaya is an Associate Director of Project Management at PCM Trials with over seven years of experience in the clinical research field. Prior to PCM Trials, she worked as a clinical research coordinator where she gained extensive knowledge of working at the site and has taken that...

Christopher J. Schaber has over 30 years of experience in the pharmaceutical and biotechnology industry. Dr. Schaber has been our President and Chief Executive Officer and a director since August 2006. He was appointed Chairman of the Board on October 8, 2009. He also serves on the board of...

I am a strategic investor that is focused on clinical stage biotech companies that are positioned to achieve a value inflection with a successful clinical trial. Prevail has invested in 50 private and public companies, which have done about $5 billion in licensing deals and had numerous successful...

I am a pediatrician with Master and PhD degree in pediatrics infectious diseases. Post-graduated in managing NGOs and Board Member certified by Board Academy.  in the last 34 years I have been managing clinical trials for academic centers and sponsored trials by pharmaceutical industries. And I...

Matthias P. Schönermark is a trained head & neck surgeon with a Ph.D. in molecular oncology. In 1998, he left his job as an Associate Professor at Hannover Medical School for a position as project leader & manager at The Boston Consulting Group, where he spent several years, supporting...

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. Her interdisciplinary and methodological research focuses on understanding what...

Denise is CEO, a board member and co-founder of Mereo. Denise has over 25 years’ experience in the biopharmaceutical industry in R&D management and venture capital. She was in R&D management at Amersham and Fisons and a senior executive at Scientific Generics before joining Rothschild...

Simone is a Senior Partner in the global lead of Simon-Kucher´s commercial Strategy Practice. She specializes in strategic pharmaceutical commercialization, launch- and brand excellence, portfolio- and lifecycle-management, including go-to-market strategies, product lifecycle strategies. She has...

Dr Sharma serves currently as the Vice President of Clinical Development as Therapeutic Head for Nephrology and Hematology at Alexion an Astra Zeneca Company. He currently serves as an independent board member for Checkpoint Therapeutics. He previously served as Chief Executive Officer for both...

Ryan Sheedy is a Dad, Rare Disease Caregiver and Founder. He lives in Bentonville, AR with his wife and 3 sons. In 2018, after 15+ years in Business Development and Fundraising he “retired” early to become a Stay at Home Dad/Caregiver for his twin sons.   One of his sons, Reynolds, was diagnosed...

Option 2  --  362 Words  &  1985 characters Professionally, Darlene is a professional accounting consultant, but her advocacy work started as a grandmother to 3 children with Hemophilia B who took a surprise, life-changing diagnosis which led to learning of gaps in emergency treatment for...

Dr. Michael Sherman serves as venture partner at RA Capital Management, a $10 billion multi-stage investment manager, dedicated to evidence-based investing in public and private healthcare and life science companies developing drugs, medical devices, diagnostics, services, and research tools. ...

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...

Amanda Singleton began her career in academia studying genetic causes of Parkinson disease and other movement disorders. After doing research at both Mayo Clinic and the National Institutes of Health, she become a laboratory genetic counselor at GeneDx. For the last 4 years, Amanda has been working ...

Rachel brings more than a decade of experience in every development phase of rare disease and cell and gene therapy (CGT) clinical trials to her work with Parexel’s rare disease clients. Previously, she served as Vice President of Clinical Operations, Portfolio Director of Rare Disease, and Global...

Laura is the Head of Insight & Advocacy at Metabolic Support UK (MSUK), the leading umbrella patient organisation for people with inherited metabolic disorders (IMDs). She leads MSUK's data insight practices and advocates on behalf of the IMD community through advisory boards, panels, task...

Oskar Smrzka brings a rich background across fundamental science and biotech industry, from rare genetic diseases to cancer. Leveraging his multidisciplinary scientific and technological expertise, he founded ABLEVIA, a Vienna/Austria-based startup, aimed at targeting drug- and vector-neutralizing...

Frank L. Sorgi, Ph.D., president and chief executive officer, founded FLAG Therapeutics, Inc., in June 2013. He brings with him over 25+ years of experience as an entrepreneurial scientist in the field of drug delivery and dosage form design and 15 years as a business executive in the biotechnology ...

Dr. Stan is a Senior Scientific Project Manager in the Therapeutic Development Branch within NCATS’ Division of Preclinical Innovation, where she manages gene therapy and small-molecule projects for rare and ultra-rare diseases, with the objective of moving therapies efficiently through...

President and co-founder MLD Foundation in 2001.  Our mission ... We C.A.R.E.® ... facilitating Compassion for families, increasing Awareness, influencing & funding Research, and promoting Education for metachromatic leukodystrophy, a very rare terminal genetic neuro-metabolic lysosomal disease ...

Rare Parent, Advocate, Filmmaker, Writer, Educator, Health Activist: Donna Sullivan is the Director of Patient Advocacy for Pathways To Trust, a 501C3 organization that educates healthcare providers about the unmet needs of rare disease and marginalized patient populations.  For the last...

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 Be Biopharma, Expansion Therapeutics, Hemab, and Aliada Therapeutics. Laura has a BS in Chemistry from Yale...

Jason Tardio is chief operating officer and joined Ovid Therapeutics in November 2019 as chief commercial officer. Prior to joining Ovid, Mr. Tardio served as vice president, head of the multiple sclerosis franchise at Novartis (NYSE: NVS). There, he was responsible for developing and managing all...

Matt Teuteberg is the Founder, President, and CEO of Splash Clinical, a global patient recruitment firm that’s pioneered the use of digital & social media to recruit patients for clinical trials. He founded Splash Clinical to help solve the problem of patient enrollment in clinical trials by...

Maria Törnsén is President North America for Calliditas Therapeutics, where she is responsible for leading the company’s US operations, including commercial and medical affairs. In addition, Ms.Törnsén is a member of the Board of Directors of Immunic Therapeutics, a role she has held since 2022. ...

Daniel Wainstock is a Patient Advocate and Researcher at Pontifical Catholic University of Rio de Janeiro (PUC-Rio). He has published several articles in renowned international scientific journals, such as The Lancet and the American Journal of Bioethics. Daniel has worked as a volunteer for Rare...

Jon Weber PhD, MBA  Senior Director Global Market Access Cell & Gene Therapy Therapeutic Area Lead Jon Weber is a Global Market Access Cell & Gene Therapy Therapeutic Area Lead at Bayer.  In this role, Jon is responsible for optimizing access for Bayer’s portfolio of Cell & Gene...

Dr. Weickert has been developing drugs at public and private companies for over 31 years. He has been CEO of Pacylex Pharmaceuticals, Fe Pharmaceuticals, illumiSonics, Sonescence, and SEA Medical Systems, CBO of Strategent Life Sciences, Corium, and Therashock, COO of Greenfire Bio and Ohm...

Patricia Weltin is the CEO/Founder of Beyond the Diagnosis, a traveling art exhibit that unites art and science to raise awareness and inspire research and innovation of treatments for people living with rare diseases. Artists paint portraits of children living with a rare disease; the portraits...

Two things light me on fire: improving patient experiences & simplifying complexity  I’ve spent nearly 20 years learning and leading patient engagement from clinical trials through product launches and sunsetting.  I created SIMPLY PATIENT to amplify the impact of life-changing drugs by...

Mr. Wilson brings more than 25 years of diversified experience in Sales, Global/US Marketing, Alliance Management, Business Development & Licensing, and General Management. Richard joined Kyowa Kirin in the summer of 2020 with responsibilities to establish the capabilities and infrastructure...

Justin Yang is a co-founder of DMV Bio, a consulting and advisory firm & co-founder of Kaibab Health, a venture creation firm based in Washington DC.

Christine Yu, MD, is the founder and president of P2P Syncro, an innovation driven medical education organization with a focus on rare disease. Dr. Yu recognized that the educational approach used in disorders commonly seen may not apply to rare diseases. She and her team are in the pursuit of...