Nicole Boice is a committed champion of families affected by rare disease. She founded Global Genes in 2008 and grew it into one of the most notable rare disease advocacy organizations focused on educating and empowering patients/advocates throughout their journey. Impact includes a membership of over 750 patient organizations, collaborations with over 100 biopharma and academic research partners, numerous support programs and educational events. RARE-X grew out of the need to provide more tools and resources for families needing to become better equipped data stewards and participants in research. A board member of several patient organizations and advisory roles within Biopharma, Nicole remains an important voice for rare disease patients globally.
Polo serves as a community-engagement specialist in the Ethical AI Initiative. He specifically assists in the development of strategies that aim to incorporate community and patient expertise into the project generally, and the advisory council specifically. Polo received his PhD and MA in the philosophy of science and bioethics from the University of Kansas. He received his BA in Philosophy, Psychology and Linguistics from the University of Texas at El Paso.
Annette Campbell is director of KCU’s campus-community pediatric health outreach program, Score 1 for Health® Campbell’s professional and scholarly pursuits have focused on building equity in health care access for all at-risk and vulnerable groups in order to improve health outcomes for individuals and to influence public health policy. Annette believes that connecting community voices to scholarly pursuits not only improves health care research initiatives, it strengthens the partnerships needed for creating authentic, sustainable health solutions. As the mother of a 22 year old diagnosed with an inherited autosomal recessive genetic disease at birth, Annette has worked for more than two decades as a patient advocate supporting continued research in order to improve quality of care and, hopefully someday, a cure for cystic fibrosis (CF).
Combining personal and professional experience as a health care leader and health equity advocate, in 2015, she initiated the first parent/family advisory council at Children’s Mercy Hospital (CMH) for CF. Since 2016, she has served on CMH’s Family Advisory Board (FAB) in order to promote the parent and community’s voice in hospital decisions and developments. Campbell has served on several national advisory committees/councils for the Cystic Fibrosis Foundation (CFF) representing the parent’s perspective and currently is a member of CFF’s Clinical Guidelines Committee. She currently is a board member of the heart-of-America chapter for CFF. In 2021, Campbell joined a team at CMH as the Lead Patient/Family Advocate for RareVoices. This grant-funded initiative aims to bring the voices of patients and families to the development of research pursuits at Children’s Mercy Research Institute. The Patient-Centered Outcomes Research Institute (PCORI) is sponsoring the initiative, and RareKC asked Campbell to contribute to this effort.
Dr. Ana Cohen is an ABMGG-certified clinical laboratory geneticist with a focus on the analysis and interpretation of genomic variants detected among pediatric rare disease patients. She completed her Masters in Biomedical Sciences at the University of Glasgow in Scotland, and her PhD in Medical Genetics at the University of British Columbia in Canada, where she described the first two patients with EED-related overgrowth (also known as Cohen-Gibson syndrome). As part of the ‘Genomic Answers for Kids’ (GA4K) research program at the Children’s Mercy Research Institute in Kansas City, Dr. Cohen wishes to make genomic testing more accessible and is currently investigating innovative strategies for diagnostic success in patients with rare diseases.
Dr. Curran holds the Donald J. Hall Eminent Scholar in Pediatric Research and he serves as the Executive Director and Chief Scientific Officer of the Children’s Research Institute, Children’s Mercy, Kansas City. He is also a Professor of Pediatrics at the University of Missouri-Kansas City School of Medicine, a Professor of Cancer Biology in the University of Kansas School of Medicine and an Adjunct Professor of Biomedical Sciences, Kansas City University of Medicine & Biosciences. Dr. Curran is responsible for defining the vision and guiding the growth of the Children’s Mercy Research Institute as a leading center for pediatric translational research.
From 1984-1995, Dr. Curran worked at the Roche Institute of Molecular Biology ultimately rising to the position of Associate Director. He then founded the Department of Developmental Neurobiology at St. Jude Children’s Research Hospital where he grew the Translational Brain Tumor Program over the period 1995-2006. He served as Deputy Scientific Director of the Children’s Hospital of Philadelphia Research Institute from 2006-2015 and he established the multi-institution Children’s Brain Tumor Tissue Consortium. Dr. Curran’s research spans the fields of cancer, signal transduction and neurobiology. He discovered and characterized the inducible Fos-Jun oncogenic transcription factor complex and demonstrated its function in diverse signal transduction processes. He also identified reelin, the gene responsible for the classic ataxic mouse mutation, reeler, and determined its role in the control of neuronal migration in the developing brain. Over the course of the last two decades, he pioneered the preclinical analysis of Hedgehog Pathway inhibitors for the treatment of pediatric medulloblastoma and transitioned this work into successful Phase I/II human clinical trials. His worked is published in over 290 papers that have been cited more than 50,000 times.
Dr. Curran was President of the American Association for Cancer Research (AACR) in 2000-2001 and he served on the National Cancer Institute Board of Scientific Advisors from 2000-2005. He was elected as a fellow of the American Association for the Advancement of Science (1994), the American Society of Microbiology (1994), the Royal Society, London (2005), the National Academy of Medicine (2009), the American Academy of Arts and Sciences (2012) and the Academy of the American Association for Cancer Research (2013). He was elected as a corresponding fellow of the Edinburgh Royal Society (2020). Dr. Curran has received several awards and honors including, the Passano Foundation Young Scientist Award in 1992, the Outstanding Achievement in Cancer Research award from the AACR in 1993, the Golgi Award from the Camillo Golgi Foundation and the Italian Academy of Neurosciences in 1994 and the Fred Epstein Lifetime Achievement Award from the Children’s Brain Tumor Foundation in 2015.
Mazen M. Dimachkie, MD, is a tenured Professor of Neurology and Director of Neuromuscular Division at the University of Kansas Medical Center (KUMC). Dr. Dimachkie is ABPN board-certified in Neurology, Clinical Neurophysiology, and Neuromuscular Medicine and holds a UCNS certificate in Clinical Neuromuscular Pathology. He is Executive Vice Chairman andamp; Vice Chairman for Research Program, Department of Neurology. He is the Associate Director of the Institute for Neurologic Discoveries at the KUMC. Dr. Dimachkie has national and international reputation as a clinician, educator and researcher.
He was recruited from the UT-Houston to lead the prolific neuromuscular Section, now Division, at the KUMC and has done superbly since 2007. He directs the Neuromuscular Medicine Fellowship which he founded at the KUMC. He directs the Tissue Biopsy Laboratory. He leads the Neuromuscular Clinics which coordinate the care of a large population of patients with muscle, nerve, neuromuscular junction and anterior horn cell disorders. In recognition of accomplishments, he was elected to be a Fellow member of the American Academy of Neurology then elected to the prestigious membership as fellow of the American Neurological Association. He serves on the Medical Advisory Board of the Myasthenia Gravis Foundation of America (MGFA) and has served on the IMACS Scientific Committee. He is recognized as a Partner of care with the MGFA.
He is a frequently invited lecturer in the fields of inflammatory muscle disorders, Pompe disease, myasthenia gravis, the Lambert-Eaton myasthenic syndrome, ALS, peripheral neuropathies and other neuromuscular topics. He has authored or co-authored 200 full-length published articles, more than 300 meeting abstracts, and 28 book chapters. His work is frequently cited by others. His Google Scholar H-index score is 36 which is outstanding.
Kimberly Fry joined our leadership team in late 2019 after her son, Charles Fry, was diagnosed with SLC6A1.
Ms. Fry brings more than 12 years of marketing and event expertise in both higher education and technology industries, currently serving as the Director of Marketing and Events at ProActive Solutions, a technology reseller in Mission, KS. She has a strong reputation for producing high end, thoughtful, creative and meaningful experiences that lead to awareness and financial support for a wide-range of audiences. Prior to ProActive Solutions Ms. Fry spent her career at the Kansas City University of Medicine and Biosciences, holding many positions, most recently as the Associate Director of University Events and Interim Director of Marketing. Kimberly attended the University of Missouri-Kansas City, receiving a Bachelors Degree in Business Administration and serving as a four year collegiate athlete as a member of the Universities Division I Volleyball team. Kimberly’s vision and goal for SLC6A1 Connect is to cure the disease, not continue to treat and live with it. She brings personal passion and determination to give her son, Charles, and all kids affected a chance to thrive and live healthy without the daily worry of this neurological disease.
In addition to Ms. Fry’s role at SLC6A1 Connect and ProActive Solutions she is a dedicated mother to son, Charles (2), daughter, Adeline (4) and wife to Nathan Fry. The family resides in Kansas City, MO.
Dee George directs legislative and regulatory efforts in the Midwest as part of the North America Public Affairs team at Novartis, a global healthcare company. In this role, she utilizes her 20+ years of state government affairs experience to demonstrate Novartis’ commitment to reimagining medicine and building trust with society.
Her focus is on furthering policies that improve patient access, protect innovation, and support the value of the biopharmaceutical industry. She collaborates with state legislators to encourage public policy platforms that reward innovation and improve patient access and affordability of medicines. She also serves as a trusted resource to state policymakers and key stakeholders, and partners with advocacy organizations on shared priorities to help amplify their messaging at state capitols.
Prior to joining Novartis, Dee was the State Government Affairs Director at the Leukemia & Lymphoma Society where she developed and managed legislative and policy strategy to raise awareness of blood cancers and drive state policy to support patient access.
Dee and her family reside in St. Paul, Minnesota where she enjoys being a hockey, baseball, dance and gymnastics Mom to her two sets of twins, as well as plays a supporting role to her husband’s small business.
Sarah Gladstone MD, is a pediatrician who completed her MD at Vanderbilt University and pediatric residency at The Children’s Hospital of Boston prior to practicing in primary care in Kansas City, MO for 15 years. She began working with WS researchers and parents to facilitate communication and help find a treatment for WS after her daughter was diagnosed with WFS1-Related Disorder in 2016. Sarah started the Unravel Wolfram Syndrome Fund in 2016 to support WS research by Dr. Fumi Urano at Washington University in St. Louis and joined forces with other WS parents to help create WSRA (the Wolfram Syndrome Research Alliance) in 2020. She is currently working with Amylyx Pharmaceuticals and Dr. Urano to initiate a clinical trial treating WS patients with AMX0035, a novel treatment for Wolfram syndrome.
Ivailo Grigorov is a Financial Advisor and special needs parent to Lucca, who is 7 years old and has a diagnosis of Prader-Willi Syndrome. After 15 years as a Store Director at Target, Lucca inspired Ivailo to change career paths and pursue special needs planning. Ivailo realized that families like ours can benefit from a comprehensive plan that can guide them through the years and can ultimately maximize a child’s overall quality of life. Ivailo’s team primarily works with families who have a loved one with disabilities that will require some support not only now but in the distant future.
There are many common mistakes in traditional planning that if not properly addressed can hinder access to programs and benefits. Ivailo’s goal is to serve as the quarter back for the family’s planning, educate on most efficient ways to save, optimize and protect our loved ones’ futures and ultimately provide a piece of mind.
Join us for this educational workshop to learn more about special needs planning. Topics covered will include an overview of SSI & SSDI, Medicaid & Waivers, ABLE accounts, special needs trusts, and other important considerations to help family members better understand how planning for a child with special needs is different both legally & financially.
Dr. Sonia M. Hall is the President & Chief Executive Officer for BioKansas, a bioscience focused non-profit committed to leading the effort of supporting and growing Kansas’ bioscience ecosystem. Dr. Hall received her PhD in Molecular, Cellular, and Developmental Biology at the University of Kansas, followed by two years of postdoctoral training at the University of Massachusetts Medical School. She has focused her career on designing and leading initiatives that aim to make science and the paths towards scientific careers more equitable. These efforts have resulted in the creation of numerous programs, publications, and initiatives, leading her to share her expertise with a variety of national and international audiences, including the Hungarian Academy of Science, Genetics Society of America, the Future of Research, government agencies, and many more.
Whether speaking with Congressional representatives, academic leadership, corporate leaders, or international scientific audiences, she has had an ongoing commitment to highlight the scientific contributions of researchers and companies in the heartland. This passion for the individuals and innovation in the Midwest led Dr. Hall back to Kansas in 2019 to lead BioKansas and bring the vision of building the region into a hub for biologics research, manufacturing, and commercialization to reality. By fostering an inclusive culture that embraces bold creativity, continuous improvement, and discovery, Dr. Hall continues to leverage the support of the bioscience network; including members, regional partners, and the BioKansas leadership team to alter the trajectory of the region’s bioscience industry.
As President and CEO of CicloMed LLC, Ms. Ham leads a team of experts committed to developing and commercializing an innovative, less-invasive approach to fighting bladder cancer. Since 2015 she has served as President and CEO of CicloMed’s parent company, BioNovus Innovations LLC, which is building a portfolio of organizations to advance scientific discoveries into transformational improvements in healthcare. She is also an angel investor. Ms. Ham started her career as a Registered Nurse in hospitals and outpatient settings, then became a clinical administrator. In 1997, she founded Ambulatory Surgery Assistance, Inc., which developed more than 30 ambulatory surgery centers. After selling that company to Nueterra Healthcare in 2005, Ms. Ham served as President of Nueterra Healthcare International. She is a Registered Nurse graduate of Saint Luke’s College of Nursing. Ms. Ham also serves as a mentor with Techstars and a board member of Operation Breakthrough.
Focused on improving health outcomes for people living with rare diseases by advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy, Annie’s work includes building strong partnerships with policy makers, federal agencies, Industry, and alliances.
Annie has served within the community for nearly three decades through her roles with Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA). In that time she helped lead legislative efforts around passage and implementation of the MD-CARE Act (2001, 2008, 2014), the Patient Focused Impact Assessment Act (PFIA) which became the Patient Experience Data provision within the 21st Century Cures Act (sec 3001), engagement with the FDA and Industry around regulatory policy and therapeutic pipelines, led access efforts as the first therapies were approved in Duchenne, and engaged with ICER around the development of the modified framework for the valuation of ultra-rare diseases.
Annie’s community roles include service on the Board of Directors of Cure SMA, the PFDD Works coalition, the Patient Driven Values in Healthcare Evaluation (PAVE) Steering Committee, FasterCures Cures for Life initiative, the National Health Council’s PCORI Valuation Group, the Innovation and Value Initiative (IVI) Patient Advisory Committee, the National Duchenne Newborn Screening Pilot Program Steering Committee, the Institute for Gene Therapies (IGT) Patient Advocacy Advisory Council, the State Rare Disease Education Initiative (STRiDE) National Steering Committee, and as a member of the NIH National Center for Advancing Translational Sciences (NCATS) Advisory Council and the Cures Accelerator Network (CAN) Advisory Board.
Michelle McMurry-Heath assumed the leadership of the Biotechnology Innovation Organization (BIO) as President and CEO on June 1, 2020. A medical doctor and molecular immunologist by training, Dr. McMurry-Heath becomes just the third chief executive to steward the world’s largest biotechnology advocacy group since BIO’s founding in 1993.
BIO represents 1,000 life sciences companies and organizations from 30 countries. The organization’s mission is to support companies that discover and deploy scientific breakthroughs that improve human health, environmental stewardship, and sustainable agriculture.
The common thread in McMurry-Heath’s work across academia, government and industry has been her focus on broadening access to scientific progress so more patients from diverse backgrounds can benefit from cutting-edge innovation. Driven by her own past family experiences navigating clinical trials and funding uncertainties within the rare disease community, McMurry-Heath calls “the distribution of scientific progress the social justice issue of our age.”
She comes to BIO from Johnson & Johnson where she served as Global Head of Evidence Generation for Medical Device Companies and then Vice President of Global External Innovation and Global Leader for Regulatory Sciences. She was also instrumental in bringing J&J’s incubator, JLabs, to Washington, DC. She led a global team of 900 with responsibilities in 150 countries around the globe.
Lindsey is a passionate global citizen that centers her life around service to others. For the world, she is committed to creating inclusive and safe spaces for everyone. She has extensive experience in program management, research, leadership, and academic writing across the non-profit, academic, for-profit, and government sectors. Lindsey holds a PhD in Therapeutic Science from the University of Kansas. As a social scientist Lindsey has worked to better understand and provide evidence-based interventions, practices, and processes to underserved communities as they interact with the numerous systems (e.g., healthcare, education, community programs) across their lifespan. For her community, Lindsey is dedicated to facilitating the connection of people to the resources they need to get the lives they want, as she involves herself in several community-based programs.
Dr. Kimminau is a leader and advocate for patient and stakeholder engagement in biomedical research, an area that is especially needed in rare disease. As a community based participatory research trained anthropologist, she has served as the co-director of community engagement for the University of Kansas’ CTSA program and as the engagement and evaluation lead for multiple, nationally funded awards. With patient partner Cheryl Jernigan, she established the first patient engagement program for the NCI-designated KU Cancer Center. She is leading the inclusion, diversity and equity efforts as well as establishing, with patient partner, Jeff Ordway, a Patient and Community Advisory Council for the new NextGen Precision Health initiative at the University of Missouri. For five years, Kimminau served as the research director of the American Academy of Family Physicians National Research Network where she championed physician engagement with training that encouraged clinic-level patient engagement in routine clinic settings. She was the principal investigator for one of only AHRQ funded Centers of Excellence in practice-based research. Among her proudest accomplishments is helping the Argentine Neighborhood Development Association successfully engage their residents and develop a business plan that brought a full-service grocery store to their food-desert community and in help launch RareKC, serving as the inaugural secretary for the organization.
Audrey Masoner is the Chief Financial Officer for the UnitedHealthcare Community Plan of Kansas. She brings a diverse background of professional leadership experience, spanning organizational disciplines from finance to operations. Before joining UnitedHealthcare, Audrey most recently served as a Senior Finance, Pricing and Proposal Manager, at CenturyLink. Prior to that, she served as Director of IT Account Management at DST and Senior Product Development Manager at Blue Cross and Blue Shield of Kansas City (Cobalt Ventures). She held multiple finance roles at Sprint, and lead divestiture and integration roles within Embarq/CenturyLink. She received a BSBA in Economics and Finance from Rockhurst University and her MBA from Baker University. Audrey is also a local children’s book author and volunteers at reading events and educational speaking opportunities on a regular basis. She currently serves on the boards of Inclusion Connections, Junior Achievement KC and Kansas City Young Audiences.
Nora McGhee, PhD, is a Senior Program Officer for the Clinical Effectiveness and Decision Science program at the Patient-Centered Outcomes Research Institute (PCORI) where she supports the Clinical Effectiveness Research priority area. She is also the staff co-lead for the PCORI Rare Disease Advisory Panel. She has previously worked in the PCORI Office of the Chief Science Officer and Merit Review. Before joining PCORI, McGhee managed the review of proposed new medical devices for several health systems. McGhee received a BA in biology from Oberlin College and a PhD in pharmacology from Duke University.
Lesa Mitchell is the Managing Director of the Techstars Kansas City Accelerator. Lesa’s career has included roles as a corporate executive, entrepreneur, and consultant to multinational corporations expanding their innovation footprints. For ten years, Lesa was VP Innovation & Networks at the Kauffman Foundation where she designed, implemented, and scaled models focused on initiating new markets and recovering markets in support of economic growth. Lesa is an advisor for Modern Meadow (NYC), CapLinked (LA), Neighborly (SF), Copia (SF). Lesa is a long time Sr. Fellow Milken Institute and is on the board of directors of the Althea Foundation in San Francisco.
Physician-scientist and rare disease expert with a record of leadership in both academic medicine and the pharmaceutical industry. Past President of the Medical Staff at Boston Children’s Hospital. Former Head of Global Medical Affairs for Rare Neurology (neurogenetic diseases) at Sanofi Genzyme. Expert in both laboratory and clinical research, with experience across all phases of drug development, and multiple successful, direct interactions with the FDA. Board certified in Pediatrics, Clinical Genetics, and Clinical Biochemical Genetics. Fellow of both the American Academy of Pediatrics and the American College of Medical Genetics and Genomics.
Eric Ochmanek has an experienced record of partnership recruitment, team building and cultivation, utilizing limited resources, government liaison, project management, and external affairs. Throughout my career I have created success at all levels from the public to private sector, from small business to Fortune 500. Working multiple projects, fast-paced, under high stress, with limited resources has been a fixed factor in almost all roles.
Dr. Pastinen has been the Director of the Genomic Medicine Center (GMC) at Children’s Mercy Kansas City since 2017. Dr. Pastinen joined Children’s Mercy from McGill University. He held the Canada Research Chair from 2007 to 2017 and has a significant track record of academic achievement. He has authored over 170 peer-reviewed publications, including papers in top-tier journals Cell and Nature Genetics. Prior to joining Children’s Mercy, he led the epigenome mapping center at McGill as part of the International Human Epigenome Consortium. Since 2019 Dr. Pastinen is leading Genomic Answers for Kids, a foundational initiative for future genomic research at Children’s Mercy Research Institute that aims to develop better tools to learn from 30,000 children with rare genetic conditions and disseminate this data resource to accelerate rare disease research globally.
Dr. Jill Peltzer is an Associate Professor at the University of Kansas School of Nursing. She teaches theory development and qualitative methods in the PhD program. Dr. Peltzer has worked as a clinical nurse specialist in pain management and internal medicine and as a staff nurse in neurology and hematology/oncology. She also spent a year in South Africa as a Peace Corps Volunteer working in HIV and substance abuse prevention. Dr. Peltzer’s research focuses on addressing health inequities among populations who have been historically excluded, using patient and community engagement methods, and translating research to policy to address the structural issues that cause and perpetuate inequities. Dr. Peltzer co-leads the Frontiers Clinical & Translational Science Institute’s Integrating Special Populations program.
Donna Pitman is a reporter and anchor at KMBC-TV in Kansas City where she’s had the privilege of sharing the stories of our neighbors for more than twenty years. An Independence native and University of Missouri graduate, Donna’s most coveted role is that of “Mom” to her three children. Their family became a part of the Rare community when they discovered, thanks to a newborn screening test, their youngest son has Pompe Disease. In 2020, his big sister was diagnosed with the rare genetic disorder as well. Donna and her husband Cody started the nonprofit “Gabriel’s Gale” to raise awareness about Pompe as well as funds for a better life for those living with it.
Matthew Pjecha, MS, is the Ethics Lead for the Ethical AI Initiative at the Center for Practical Bioethics, a community-based project developing best practices and recommendations for the use of artificial intelligence systems in healthcare. His research focuses on the intersection of healthcare, policy, and emerging technology. Matthew received a B.A. in Philosophy from the University of Missouri – Kansas City where he focused on bioethics and philosophy of science. He then completed an M.S. in Public Policy from the Georgia Institute of Technology focused on health policy and a graduate certificate in science, technology, and society studies.
Kelly Ranallo is a healthcare business entrepreneur, long-term parent advocate and the Founder/President of the Turner Syndrome Global Alliance (TSGA). She has a teenage daughter who was diagnosed with Turner syndrome (TS) in 2006 and was inspired to join the nonprofit world to advocate for girls, women and families with TS. In partnership with Children’s Mercy Kansas City through her position on the Family Advisory Board (FAB), Kelly set about building a Turner Syndrome Clinic from within the hospital to address the unmet needs of families in and around the Midwest. The Great HeighTS Clinic opened in 2010 and has since become a national model for the development of 38 pediatric-based clinics across the country. In July of 2018, the TSGA went on to partner with the University of Kansas Hospital to launch the first of its kind adult TS specialty clinic to advance the transition of care and research development across the lifespan.
Dennis Ridenour became President and Chief Executive Officer of BioNexus KC on February 18th, 2019. Prior to joining BioNexus KC, Mr. Ridenour was President and Chief Executive Officer of BioKansas, a regional life science & healthcare trade association based in Fairway, Kansas, where he managed all facets of the organization, including day-to-day operations, member development and engagement, strategic planning, and finance. Prior to joining BioKansas, Mr. Ridenour worked for 6 years at the Stowers Institute for Medical Research in Kansas City, MO, and at Merck Research Labs in Boston, MA, for 3 years. Mr. Ridenour started his professional career as a researcher in the Department of Neurobiology at Harvard Medical School. Dennis earned both a B.S. in Biological Sciences (1999) and an M.S. in Veterinary & Biomedical Sciences (2002) from the University of Nebraska-Lincoln, as well as an M.B.A. from the University of Kansas (2012).
Special needs parent and mom to Hudsyn, her daughter who has complex medical needs, Kacy is a financial advisor in Kansas City. After six years in the financial services industry, she opened her practice with Northwestern Mutual in 2013 because she had difficulty finding good, sound advice from a variety of financial advisors who could help her develop a solid plan for her daughter. Her purpose in life is to awaken understanding and help others become empathetically aware of people with disabilities. She focuses on helping special needs families with proper financial planning throughout their lifetimes. Her book, “Every Now & Then, I Fall Apart: A Mother’s Memoir of Life After a Traumatic Birth” is scheduled to be published in 2022.
Dionne L. Stalling is a thoughtful leader, speaker and patient advocate who has dedicated her life to raise awareness of chronic, invisible and rare diseases. She is steadfast to ensure equitable representation and inclusion for Black people in the rare disease community. In 2020, Dionne founded the nonprofit organization Rare And Black where she currently serves as its Executive Director. As a chronically ill patient who proudly lives with five rare diseases, Dionne believes that her purpose in life is to serve as a mouthpiece for those who cannot speak for themselves; and to do it in a way that is meaningful and compelling. Dionne is a Washington University Community Research Fellow. She is a member of the Washington University Patient Engagement Advisory Board as well as the Washington University Patient Research Advisory Board. Dionne often speaks as an advocate to medical organizations, pharmaceutical companies and to congressional members on Capitol Hill to change policies and systems to improve health inequities as well as close the gap on health disparities. Dionne is the host of the new podcast Living Rare And Aware where she hopes to enlighten and inspire its audience about living with rare diseases. Her forthcoming memoir, I Won’t Complain is due to be published in August 2022.
Jay Vivian is a Faculty Researcher at Children’s Mercy Research Institute at Children’s Mercy Hospital in Kansas City and serves as the Director of the Rare Disease Model Research Program. A major focus of his lab involves the development and analysis of animal and pluripotent stem cell models of rare pediatric neurological disorders, using genetic data mapped by human genetics studies. These collaborative translational efforts make substantial use of genome editing to engineer genetically modified mice and patient-specific induced pluripotent stem cells for therapeutic discovery. A native of Illinois, he obtained his PhD in developmental genetics at the University of Texas M.D. Anderson Cancer Center. He currently serves on the Board of Directors of multiple rare disease foundations including RareKC, the Mowat-Wilson Syndrome Foundation, and the Jansen-deVries Syndrome Foundation.
Teneasha Washington, PhD, MPH, is the Founder of The Washington Group, a consulting agency, that focuses on addressing client’s needs in a variety of specialty areas. Her current research focuses on serving as the lead of community engagement efforts for the Mindfulness, Exercise, Nutrition, to Optimize Resilience (MENTOR) program. In this role, she develops micro-funding opportunities for local communities to implement programs relevant to their needs. In addition, she develops and provides in-person and online trainings focused on community engagement, program implementation, and building sustainable partnerships among communities, non-profits, and academia. She currently serves as the lead of diversity initiatives at RARE-X. In addition, she is a QM-certified professor teaching undergraduate and graduate students in the UAB School of Public Health on community organizing, social and behavioral sciences, intervention development, and research methods. Washington holds degrees from UAB (BS, Biology; MPH, Public Health; PhD, Health Education/Health Promotion, Public Health; Present MBA, expected graduation summer 2022).
Sara Hart Weir serves as the C.E.O. of RareKC and has over two decades of experience working in the nonprofit sector – nationally, regionally and locally. Most recently, the 2020 Primary runner-up in Kansas’ Third U.S. Congressional district in August 2020. Prior to running for office, Weir was the President & C.E.O. of the National Down Syndrome Society (NDSS).
Weir’s life has been about leading differently, serving others and delivering results with the leadership philosophy of “if you’re not at the table, you are on the menu”. Sara deploys an “in the trenches” leadership style by engaging directly with constituents – in local communities, in the classroom, on the job site or around kitchen tables. Weir’s passion for “giving a voice to the voiceless” and ensuring underserved and under-represented populations have a seat at the table was inspired by Kasey, her best friend of twenty years who had Down syndrome.
Weir’s proudest accomplish to date is leading the bipartisan advocacy effort behind the passage of the landmark, historical Stephen Beck Jr. Achieving a Better Life Experience (ABLE) Act in 2014 (P.L. 113-295). The tax legislation garnered the support of 381 of 435 US Reps. and 78 of 100 US Sens. (one of the most bipartisan laws of all time). Subsequently, oversaw the effort to enact 49 state ABLE laws as well as played a significant role in working to launch over 40 state ABLE 529 Plans with State Treasurers. The ABLE Act established tax-free savings accounts for people with disabilities and is hailed as the most significant legislation for the disability community since the Americans with Disabilities Act (ADA).
Dr. Scott J. Weir, PharmD, PhD, is Director, Institute for Advancing Medical Innovation (IAMI), University of Kansas Medical Center (KUMC). He also serves as the Associate Director for Translational Research for the University of Kansas Cancer Center. Weir holds academic appointments as Professor in the departments of Cancer Biology and Pharmacology, Toxicology and Therapeutics as well as the Department of Medicine, Division of Medical Oncology at KUMC. He is the Kelly Family Foundation IAMI Professor in Cancer Drug Discovery and Development. IAMI conducts product development-focused translational research, transforming laboratory and bedside discoveries into medical innovation products with clear paths to market. IAMI’s areas of focus are drugs, diagnostics and medical devices that benefit pediatric, adolescent, adult and elderly patients suffering from cancer and rare diseases.
Nicole, the Global Liver Institute’s Pediatric and Rare Liver Diseases Program Manager, is driven by her 13 years of experience in the liver community and as an autoimmune / transplant patient and caregiver. After earning her BA in English and Education, she taught on the island of St. Thomas, US Virgin Islands, and in rural Kentucky. Nicole also holds an MA in English: Rhetoric and Composition. Her interests include policy, access, equity, environmentalism, and education. As a volunteer, Nicole is very active in the Young Adult Representatives of RDLA (YARR) and serves as a member of the Speakers Bureau.
