Mrs. Milligan is Executive Director at the Barth Syndrome Foundation.  She has dedicated her career to improving the lives of children and their families through scientific advancements and social equality. Trained in public health and international relations, Emily brings years of experience managing research portfolios and innovating business models in the nonprofit sector. Previously, she was a vital contributor at Columbia University where she was a founding member of the Center for Social Inequalities and Health.  She went on to join JDRF (formerly known as the Juvenile Diabetes Research Foundation) where she headed the research operations and scientific teams and oversaw an average annual $100 million research portfolio.  More recently, Emily launched the T1D Fund, an $80 million, mission-driven venture fund that invests in companies developing life-saving products for individuals living with type one diabetes. She has been a guest speaker at the U.S. Food and Drug Administration (FDA), National Institutes of Health (NIH), and co-authored guidance documents on patient-focused drug development standards. Emily is passionate about finding cures for rare diseases.

Ms. Gordon is President and Executive Director at The Progeria Research Foundation, and a well-known leader in the rare disease community.  Under her leadership, The Progeria Research Foundation funded and co-coordinated the clinical trial that was the catalyst for the FDA approval of Zokinvy, a life-extending drug for Progeria.  The organization has received Research!America’s 2014 Paul G. Rogers Distinguished Organization Advocacy Award for bringing progeria from obscurity to the forefront of successful translational research.  Ms. Gordon is a graduate of Tufts University and Northeastern University School of Law, and is a licensed attorney with extensive experience in civil litigation.

Ms. Davis is Founder & President of Texas Rare Alliance & Member, Texas Newborn Screening Advisory Committee Member & Rare Disease Subcommittee Chair. She joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1. Khrystal and her husband worked with relentless urgency to help develop and secure a treatment for Hunter. In eight short weeks, Hunter was the first SMA patient to receive a disease modifying treatment. Since then, she has been advocating tirelessly for rare disease research and for patients in need: She has been an invited witness at the US House of Representatives, a panelist at BIO multiple times, a speaker at the Global Genes Patient Advocacy Summit, a presenter at THBI Texas Capitol Briefing on Gene Therapies, and more. Ms. Davis earned her Juris Doctor from Stetson University College of Law; and certifications in Design and Interpretation of Clinical Trials, and Drug discovery.

Doug Lindsay’s life story became CNN’s top online story of 2019.  At 21 a debilitating autonomic-adrenal condition that confounded doctors left him homebound and bedbound, confined to hospital bed 22 hours a day for the next 11 years.  During that time, he discovered what was wrong, developed novel treatments to keep his rare condition at bay, and eventually developed the surgery used to fix him.  Now Doug Lindsay is a standing ovation TEDx speaker and a Personal Medical Consultant who works one on one with a small number of rare disease and complex condition patients.  Doug is chair of PCORI’s Rare Disease Advisory Panel, co-chair of Washington University’s Community Advisory Board for the Institute for Clinical and Translational Research, an advisor to the NIH’s ACTIV-1 IM COVID therapeutics trial, and a two-time ePatient Scholar at Stanford Medicine X.  In his advisory work with The 40%, Doug hopes to change the way rare diseases are researched, funded, and treated.

Dr. Wang is Chief Executive Officer of Refuge Biotech, a cancer therapy company he co-founded following nearly a decade of life science investment banking experience. He most recently served as director of healthcare investment banking at Barclays Capital, where he served as strategic financial advisor and helped raise capital for companies in the healthcare industry.  Dr. Wang is a survivor of a rare form of cancer who is passionate about bringing forth novel medicines that will transform rare disease and cancer care.  He earned an MBA from Columbia Business School and a Ph.D. in Electrical Engineering from Princeton University.

Dr. Harlan is the former Head of the Diabetes Branch of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the National Institutes of Health (NIH), where he oversaw every research project on diabetes funded by the NIH.  He is currently Chief of Diabetes Division and Co-Director of the Diabetes Center of Excellence at the University of Massachusetts Medical School.  As an expert experienced in research grant management, Dr. Harlan understands the power of patient organizations in driving medical research and new therapy development.  His experience with managing diabetes research at the national level is readily translatable to rare disease research.