Tracy Antonelli is a graduate of Simmons College and the Boston University School of Public Health. Tracy was a clinical researcher at New England Research Institutes and Boston Children’s Hospital. She contributed to numerous groundbreaking studies, including the Stroke Prevention Trial in Sickle Cell Anemia. Tracy’s work has been published in The New England Journal of Medicine. Tracy became a stay at home Mom in 2012, when she and husband Patrick Mooty adopted their first daughter (Emmie) from China. They have since adopted daughters Rosie and Frannie from China. Tracy, Emmie, Rosie and Frannie have transfusion-dependent thalassemia. All of them need blood transfusions every 3 weeks and daily chelation therapy. Tracy is a Board Member for the Cooley’s Anemia Foundation and a frequent patient speaker for Harvard Medical School. She advocates for blood donation and volunteers for Boston Children’s Hospital. Emmie, Rosie and Frannie are Children’s Miracle Network Hospitals Champions for 2022. While living with a chronic blood disorder is a big part of their lives, they are an active and fun-loving family, thriving with thalassemia.

As President and CEO of the Massachusetts Biotechnology Council, Bob’s mission is to advance Massachusetts' leadership in the life sciences to grow the industry, add value to the healthcare system and improve patient lives. Over the last decade, Bob has truly become a champion for patients by ensuring innovative companies have the best environment possible to research, develop, and commercialize breakthrough therapies and cures for people around the world who need and deserve them. Bob has spent his career in both the public and private sectors. Before joining MassBio, he served as the Undersecretary of Economic Development within Governor Deval Patrick's administration, where he prioritized both healthcare and economic development issues and was a strong advocate for the life sciences industry in Massachusetts. Prior to that, he was elected as State Representative to the 11th Norfolk district for three terms. Bob has also held senior executive positions in the environmental services, capital management and venture capital industries. He brings passion and energy to all of his endeavors.

Mark Dant is the current Chairman of the Board of the Washington DC based EveryLife Foundation for Rare Diseases, a science-based advocacy organization dedicated to accelerating biotech innovations for rare disease treatments through science-driven public policy. Mark is also the founder and Volunteer Executive Director of the Ryan Foundation and former President and CEO of the National MPS Society. For the past 25+ years, Mark and the Ryan Foundation have partnered with numerous research scientists and universities to help innovative projects move toward treatment in lysosomal storage disease. Mark and his family have been key advocates speaking to the FDA and in 2009 successfully championed the US House of Representatives to pass the Ryan Dant Health Care Opportunity Act, a bill designed to help those living on Medicaid assistance become gainfully employed. The Dant’s journey has been documented on CBS 60 Minutes, CNN, Biography Magazine, Readers Digest in 13 languages around the world, Golf Digest, the LA Times and numerous newspapers and news outlets across the US. Mark retired from police work in 2016 as Assistant Chief of Police with the Carrollton Texas Police Department after serving 32 years as a Patrol Officer, Detective, and Commander leading multiple divisions and Bureaus to include Patrol, Criminal Investigations, Intel, and SWAT. Mark spends his time now volunteering for the EveryLife Foundation, the Ryan Foundation, and numerous other rare disease organizations to help empower the patient advocate through the understanding that all of us have the power to turn action to hope and hope to reality.

Basil T. Darras, MD, is the Joseph J. Volpe Professor of Neurology at Harvard Medical School. At Boston Children’s Hospital, Dr. Darras is Associate Neurologist-in-Chief and Chief of the Division of Clinical Neurology. He is Director of Boston Children’s Neuromuscular Program, which includes one of the oldest and most active Muscular Dystrophy Association clinics in the country. Dr. Darras is a pediatric neurologist with advanced training and certification in human genetics and neuromuscular medicine. His primary research focus is in the field of pediatric neuromuscular disorders. His specific research interests and major publications have focused on the molecular genetics, diagnostics, and therapeutics of Duchenne muscular dystrophy, Becker muscular dystrophy, spinal muscular atrophy (SMA), and other myopathies, and on defining the indications for new diagnostic methodologies in the evaluation of children with pediatric neuromuscular diseases. His clinical focus is the care of children with neuromuscular conditions originating from inherited or acquired conditions of the motor unit. He is Principal Investigator for the Boston Children’s site of NIH’s NeuroNEXT clinical research network, for the SMA Foundation’s Spinal Muscular Atrophy PNCR Network, and for trials testing novel treatments for SMA, including the clinical trials for Spinraza® and Zolgensma®, recently approved by the FDA as the first treatments for SMA. He is frequently invited to lecture at national and international meetings on topics related to his expertise in neuromuscular disorders and genetics. He has published over 190 original reports in peer-reviewed journals and over 80 chapters, reviews and editorials, and is Editor-in-Chief for the second edition of Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach, published by Elsevier in 2015.

Khrystal K. Davis Founder & President of Texas Rare Alliance & Texas Newborn Screening Advisory Committee Member. Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo trials, provide a means of access for the weakest SMA patients, accelerate the approval, and approve the treatment for all SMA patients regardless of age or type of SMA. In an interim look completed on August 1, 2016, the FDA found the treatment met trial objectives. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016. Khrystal is committed to improving health outcomes in those with rare diseases through improved access to rare disease diagnoses and treatments. She advocates for whole genome sequencing of newborns, the expansion of newborn screening programs, insurance policies that conform to FDA labels for orphan drugs, and clinical trials and real-world evidence that include endpoints and data points that are meaningful to patients and caregivers. She is a proponent of patient-driven access to rare disease treatments. Khrystal holds a Juris Doctorate from Stetson University College of Law and is certified in Clinical Trial Design and Interpretation by Johns Hopkins University and Drug Discovery by University of California San Diego through the Coursera program.

Barbara Henry has nearly thirty years of managed care experience and is currently the Manager of Clinical Pharmacy Services at Harvard Pilgrim Health Care serving members in Massachusetts, Maine, New Hampshire and Connecticut. In this role her responsibilities focus on the development and management of multiple formularies, including drug tier placement and utilization management programs. She also provides leadership in the creation and execution of pharmacy strategy including comprehensive drug cost management initiatives, innovative payment models and monitoring medical and pharmacy drug trends. Works closely with a cross-functional team to implement quality programs with a patient centric and value based approach.

Michelle began working at Ra Pharma in 2011 as an assay development scientist in the Target Biology group. Michelle’s initial role in the company was to design and perform the cell-based and biochemical assays needed for all projects. As Ra Pharma grew and identified a lead project, Michelle’s role grew to developing the pharmacodynamic assays needed for the animal studies. She then went on to design and oversee the validation of these assays for use in human clinical trials. Michelle’s career at Ra Pharma transitioned into the Translational Sciences group and now into the Clinical Development group where she is the Senior Manager for Clinical Science & Biomarker Development. Michelle is currently responsible for the pharmacodynamic, pharmacokinetic, pharmacogenomic and biomarker data across all completed and current Phase 1, Phase 2 and Phase 3 clinical trials at Ra Pharma for their C5 inhibitor, zilucoplan.

Kollet is responsible for project leadership, analysis and the development of NHF’s healthcare payer education strategies, policies and programs, including the Comprehensive Care Sustainability Collaborative (CCSC). Prior to her tenure at NHF, Kollet served as the Executive Director of the Bleeding & Clotting Disorders Institute in Peoria IL, a federally recognized Hemophilia Treatment Center(HTC) of Excellence, where she developed and grew an inhouse specialty pharmacy program that became one of the larger programs in the country. Kollet has also served on the Hemophilia Alliance Executive Board of Directors, served as the chair of the national payer subcommittee and sat on the Illinois Hemophilia Advisory board. With hemophilia ranking #12 of 20 for highest cost catastrophic claims, #9 for reported high cost claims for stop loss carriers and #8 of 10 highest cost claims for jumbo employer sponsored health plans ; Kollet strongly believes that the stage is set for all parties who share in the cost risks associated with rare chronic disorders, to change their current practice of giving away their healthcare brainpower to those who might have experience with specialty pharmacy dispensation for these chronic rare diseases, without at least entering into dialogue with those that are the experts in managing this patient population.

Tara Lavelle is an Assistant Professor at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies and an Investigator at the Center for the Evaluation of Value and Risk in Health. Her research addresses a range of topics related to the cost and value of health care services delivered in the United States. Dr. Lavelle is particularly interested in advancing the use of comparative and cost-effectiveness research in vulnerable populations, including children and individuals with mental illness. Previous work includes the development of novel methods for evaluating health and economic outcomes of childhood illness, and assessing preference-based quality of life and work productivity outcomes in caregivers. Dr. Lavelle earned her PhD in Health Policy with a concentration in Decision Science from Harvard University in 2012.

During her 35-year career in medicine, Freda Lewis-Hall has been on the frontlines of health care from the standpoints of a clinician, a researcher, and a leader in the biopharmaceuticals and life sciences industries. The common thread throughout has been her passion to advocate for health equity and improved outcomes for all patients. Trained as a psychiatrist, Dr. Lewis-Hall began her medical career in patient care and became well known for her work on the effects of mental illness on families and communities and on issues of health care disparities. She has held positions of leadership at the Howard University Hospital and College of Medicine, Vertex, Bristol-Myers Squibb, Eli Lilly and Pfizer. Some of her many achievements include founding the Lilly Center for Women's Health in the 1990s; serving on the board of the U.S. Patient-Centered Outcomes Research Institute (PCORI) since its inception in 2010; creating Pfizer's public health information program Get Healthy Stay Healthy in 2012; launching the industry's first public compassionate access request portal, PfizerCARES, in 2015; and spearheading the creation of SpringWorks Therapeutics, a new company working to develop promising new treatments in underserved areas of urgent medical need, in 2017. From 2009 to 2018, Lewis-Hall served as Pfizer's Chief Medical Officer, responsible for the safe, effective and appropriate use of Pfizer's medicines and vaccines, and in this role she reshaped Pfizer's medical policies and practices to intensify the company's focus on patient engagement and inclusion. In her current role as Chief Patient Officer of Pfizer, Lewis-Hall is working to extend the reach of Pfizer's patient-facing health information and education and amplify the voice of the patient inside and outside Pfizer.

Ms. Pitluck is the Head of Global Pricing & Reimbursement (P&R) at Spark Therapeutics, Inc., a fully integrated gene therapy company committed to meeting the needs of patients living with genetic diseases. In her current role, Ms. Pitluck priced the first US Food and Drug Administration-approved gene therapy for a genetic disease, LUXTURNA® (voretigene neparvovec-rzyl). She also led the roll-out of Spark’s unique alternate payment options and outcomes-based arrangements for LUXTURNA. She is now working to develop programs to help ensure timely patient access to future one-time gene therapies in development. Prior to joining Spark, Ms. Pitluck was the Executive Director for Global P&R at Alexion Pharmaceuticals, leading all global P&R efforts, including dossier preparation and negotiations for Alexion globally. Ms. Pitluck was formerly the Director of Genentech’s Coverage & Reimbursement Policy and responsible for all US public payer policy and reimbursement issues for Genentech’s products. She also worked at healthcare consulting companies including Avalere Health and Covance Health Economics and Outcomes Services Inc., focusing on policy and reimbursement analyses for a variety of pharmaceutical, biotechnology, and medical device products related to care settings and disease areas. Ms. Pitluck received her Masters of Science at the London School of Economics and Political Science and her Bachelor of Arts from Washington University in St. Louis.

Dr. Kim Stephens is the president of Project Alive, a nonprofit organization that is a powerful voice for children and adults with Hunter Syndrome, bringing together families and advocates with researchers, industry, and regulators. Having a son with Hunter Syndrome herself, this is also a personal mission for Kim. She frequently speaks as a rare disease advocate at conferences and events and serves as a mentor and resource for newly-diagnosed families. Kim is also the founder of Inclusive Thinking – a consulting company dedicated to increasing diversity and inclusion through research, education, and strategic planning. Before founding Inclusive Thinking, she had a twenty-year career with IBM, most recently as the Global Diversity & Inclusion Communication and Education Lead. She has spoken at conferences around the world and conducted workshops on unconscious bias, cultural awareness, women's leadership, accessibility and other topics. In addition, Kim is an Associate Professor at Lincoln Memorial University teaching management and leadership in the MBA program. Kim earned her doctorate in Business from Georgia State University and focused her dissertation on implicit bias and the role of transformational conversation and social identity on behavioral change.

Glenda was diagnosed with myasthenia gravis (MG) in 2013. In 2014, she became an active volunteer member of the New England Myasthenia Gravis Support Group and continues to be an advocate for the MG community. Glenda is the New England MG Support Leader, is on the governor of Massachusetts Rare Disease Advisory Council and is a patient voice on three pharmaceutical companies doing research in MG. Glenda is a strong MG advocate, sharing her story as guest speaker at pharmaceutical companies’ events, the MGFA 2023 National Patient Conference, the 2021 American Association of Neuromuscular & Electrodiagnostic Medica (AANEM) to name a few. Her MG story has been featured in various newspapers, magazines, social media outlets and talk shows, including the Boston Globe 2023 and Biotech Week, Scripps News, Authority Magazine, and the MetroWest Daily News. Glenda is also a strong patient advocate. She is the co-leader of the Beth Israel Deaconess Medical Center Patient and Family Advisory Council co-leader. Glenda enjoys working with elderly and disable individuals by helping them with their day-to-day finances as a Daily Money Manager. She enjoys traveling, swimming, snow shoeing and entertaining family and friends.

Rehan Verjee is a co-founder and the CEO of a stealth start-up focused on enabling precision medicine through a next generation liquid biopsy platform. Previously, Rehan served as President of EMD Serono and as the Global Head of the Innovative Medicine Franchises for Merck KGaA where he led the US and global specialty medicine business through a period of significant transformation and growth that included four FDA approvals for three new medicines across cancer and auto-immune disease and a multi-billion USD deal for an EMD Serono pipeline prospect in immuno-oncology. Prior to this role, he served as Executive Vice President, Chief Marketing and Strategy officer for Merck Healthcare, Merck KGaA. Rehan is currently a member of the board of directors of Tyra Biosciences and the Massachusetts Biotechnology Council. He holds a master’s degree in Molecular and Cellular Biochemistry from the University of Oxford in the U.K.

Matt is committed to the development and commercialization of innovative therapies for rare and hard to treat neurological diseases Matt has spent more than 5 years at Biogen now. Since February 2018, He has served as VP, head of the Rare Disease Division. Prior to that Matt was Sr. Director, Chief of Staff to the Global CEO Michel Vounatsos. He also worked in Payer and Channel Marketing for both the Rare Disease and MS Franchises while at Biogen. Matt worked in Life Science Consulting prior to coming to Biogen at a number of different companies. He worked on projects with small biotech as well as large pharmaceutical companies.