Rare Disease Summit Speakers

Andrea Atherton

Andrea Atherton, Regional Medical Director, Horizon

Andrea Atherton is a board-certified genetic counselor who currently works for Horizon Therapeutics as a Regional Medical Director in Rare Diseases where her primary focus is centered around scientific education and dissemination of information on the medical management and treatment for Cystinosis. Andrea started her career 20 years ago as a genetic counselor at Children’s Mercy Hospital after graduating from the University of Texas Health Science Center with a master’s degree in genetic counseling. While working as a genetic counselor, Andrea was extremely involved in the care of pediatric and adult patients with inborn errors of metabolism, lysosomal storage disorders, mitochondrial conditions, neuromuscular disorders, neurogenetic diseases and became a huge advocate for newborn screening at the state and federal level. She served as a member of the Newborn Screening Translational Research Network steering committee and was recently appointed as the newest board member to RareKC in 2023.

On a personal note, Andrea has been married to her husband for over 20 years and they have four boys together. Their oldest son is a freshman at Crowder College where he is studying business and playing baseball for the Roughriders. Their youngest three boys are identical triplets who are sophomores in high school. Andrea volunteers her time and serves on the executive committee of the athletic booster at Lee’s Summit North High School.

Lauren Bartik

Lauren Bartik, M.S., CGC, Assistant Program Director at KUMC Genetic Counseling Program

Lauren Bartik, M.S., CGC, is the Assistant Director of the genetic counseling master’s degree program at the University of Kansas Medical Center. As a certified genetic counselor, Lauren has practiced at Children’s Mercy Hospital for over eleven years. Her primary specialty has been pediatrics with a focus on cardiology, craniofacial, inpatient consults and 22q11.2 deletion/duplication syndrome.

Blake Benton

Blake Benton, Cofounders, Coming Together for a Cure

Dedicated to expanding access to stem cell therapy for Duchenne Muscular Dystrophy and rare diseases. In 2008, Blake’s brother Ryan became the first person with DMD to be successfully treated thanks to mesenchymal stem cells. This inspired Blake to drive broader adoption of this therapy.

In 2014, while completing his degree at K-State, Blake and Ryan established CTFAC, a 501c3 nonprofit. Blake then helped Ryan as his Primary Caregiver for 5+ years while advancing CTFAC’s mission. Over the past decade, CTFAC has supported over 300 families globally seeking therapy, organized annual fundraisers that attract 1,000+ attendees, and formed partnerships with industry leaders. Recently, CTFAC finished their feature documentary, which is part of an extensive global advocacy campaign. In 2024, CTFAC will serve as the primary charity partner for the first large-scale mesenchymal stem cell clinical trial for Duchenne in the US. Blake’s background in enterprise software, strategic planning, and firsthand understanding of challenges facing the rare disease equips him to help pioneer solutions to make this therapy accessible and affordable while always putting the patient’s needs first.

Courtney Berrios

Courtney Berrios, MSc, ScM, CGC, Senior Genetic Counselor, Children’s Mercy Kansas City and Research Assistant Professor, the University of Missouri Kansas City

Courtney received her master’s in Genetic Counseling from the Johns Hopkins University / National Human Genome Research Institute Genetic Counseling Training Program and has completed additional training in clinical research and pediatric bioethics. She conducts studies on ethical and social research topics for the Genomic Answers for Kids pediatric genomic research repository. Courtney developed the Rare Voices project to inform community-centered agenda for ethics and social research in Genomic Answers for Kids and throughout our region.

Mindy Bridges

Dr. Mindy Bridges, Assistant Professor, Hearing and Speech, University of Kansas

An associate professor in the Department of Hearing and Speech, Mindy Sittner Bridges, Ph.D., CCC-SLP, joined the faculty of the University of Kansas in 2017. She teaches courses related to reading development and disorders as well as evidence-based practice.Bridges received her bachelor’s degrees in speech-language pathology and audiology and linguistics from the University of Iowa and her master’s degree in speech-language pathology from the University of Nebraska-Lincoln. She spent the next seven years working as a speech-language pathologist in a variety of settings, with a particular emphasis on pediatric language and reading development and disorders. Bridges completed her doctorate at the University of Kansas in 2009. Prior to joining the faculty at KU Medical Center, Bridges held a research position affiliated with the Life Span Institute at the University of Kansas in Lawrence.

Bridges has pursued two primary lines of research. One broad area involves the early identification of students at risk for reading disabilities. This research includes longitudinal work examining early predictors of later reading comprehension as well as the development of early identification assessments. The second area of research includes the development and efficacy testing of reading intervention provided in a classroom setting for preschool and elementary students. Both lines of research come directly from an interest in the relationship between language and reading development as well as previous work as a speech-language pathologist.

Dr. Tom Curran

Dr. Tom Curran, PhD, FRS Senior Vice President, Executive Director and Chief Scientific Officer, Children's Mercy Research Institute

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.

Michelle Davis

Michelle Davis, Executive Director, IFOPA

Michelle Davis joined the 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 as Executive Director of Nonprofit Connect. In that role, Michelle was exposed to many deserving causes, but her passion has always been in working with patient advocacy organizations, including tenures at the National Kidney Foundation and Polycystic Kidney Disease (PKD) Foundation. While at the PKD Foundation, Michelle became acquainted with the rare disease community which is part of what drew her to the IFOPA. At the PKD Foundation, Michelle served in the role of Chief Development Officer where she worked in all aspects of fundraising and maintained the organization’s relationships with the industry. Michelle also led the organization’s education and advocacy initiatives, including organizing patients for an FDA Advisory Committee meeting for the review of the first-ever treatment for PKD, Jynarque. At the IFOPA, Michelle leads the staff team and partners with the Board of Directors to execute the organization’s strategic plan. She works closely with academic and industry researchers on real-world evidence programs and clinical studies and trials. She also works on awareness programs for health care professionals and advocacy initiatives.

Kimberly Fry, Vice President of SlC6A1 Connect

Kimberly Fry, Vice President, SLC6A1 Connect

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.

Terrence Gallagher

Terrence Gallagher, Co-chair, Children’s Mercy Research Institute (CMRI) Community Advisory Board,

Terrence Gallagher is a dedicated community volunteer, focused on advocacy for children with special needs and their families. His late daughter Elizabeth had multiple medical and developmental complications related to Angelman Syndrome and another undiagnosed genetic disorder. The current co-chair of the Children’s Mercy Research Institute (CMRI) Community Advisory Board, he served throughout the Rare Voices Project in several capacities including moderating listening sessions and research question development. Terrence serves on the Children’s Mercy Family Advisory Board in addition to numerous committees and efforts related to patient experience, quality and safety, and staff education. He also served on several advisory committees for the Children’s Hospital Association and the Family Advisory Council for the Kansas Department of Health and Environment (KDHE).

An architect by profession, Terrence is a member of the Prairie Village KS City Council, the Family Advisory Council at Ronald McDonald House Charities of Kansas City and is a lifelong volunteer for the Boy Scouts of America.

Dr. Keith Gary

Dr. Keith Gary, Vice President, Mission Acceleration, The ALS Association

Dr. Keith Gary is the Vice President of the Kansas City Life Sciences Institute and on the Board of Directors at the ALS Association. He earned his Bachelor of Science degree in Biology from Central Methodist College, followed by his PhD in Biochemistry from the University of Missouri Kansas City. He completed his postdoctoral studies in Neuropsychopharmacology at the University of Pennsylvania. Dr. Gary spent a year as a Research Assistant Professor in Psychiatry at Dartmouth Medical School and four years as an Assistant Professor of Psychiatry and joint appointment in Pharmacology at the University of Connecticut Health Center. He then served as Vice President of BioNexus KC for eighteen years. In 2021, Dr. Keith Gary joined the ALS association as the Vice President Mission Acceleration.

Lauren Harrell

Lauren Harrell, PhD candidate in the lab of Dr. Gerald Wyckoff

Lauren Harrell is a PhD candidate in the lab of Dr. Gerald Wyckoff. She graduated from Emporia State university in 2017 with a Bachelors of Science in Biochemistry and Molecular Biology and a minor in Mathematics. She then earned her Masters of Science in Cell and Molecular Biology in 2019 from the University of Missouri-Kansas City, before transitioned into the interdisciplinary PhD program to begin work on her dissertation in the Wyckoff Lab.

During her undergraduate studies, Lauren became interested in personalized medicine and developing tools to make the healthcare experience less cumbersome for both patients and providers. This early interest has profoundly influenced her research focus, which centers on harnessing existing big data platforms. She employs a blend of cutting-edge techniques, such as machine learning, natural language processing, and genomic studies, to unveil patterns in diseases traditionally regarded as challenging to diagnose.

Bryce Heese

Dr. Bryce Heese, Chief of Genetics, Children’s Mercy Kansas City

Dr. Bryce A. Heese is a clinical geneticist in Kansas City, Missouri and is the Chief of Genetics Children’s Mercy Kansas City Hospital. He received his medical degree from University of Nebraska College of Medicine and has been in practice for more than 20 years. He completed his residency in Genetics at the Mayo Clinic in Rochester, MN and Pediatrics at Creighton University/University of Nebraska Medical Center in Omaha, NE.

Michele Herndon

Michele Herndon, MSN, RN, Program Director, Undiagnosed Diseases Network Foundation's Patient Navigation Program

Michele Herndon is the Program Director of the Undiagnosed Diseases Network Foundation’s Patient Navigation Program. For the past two decades, she has served as a pediatric nurse, leader, and manager in an academic hospital setting. Michele is also the mother to Mitchell who enrolled in the UDN in 2017 after five years of symptoms. After genetic sequencing and a model organism study using fruit flies, his gene mutation was identified by the UDN. Mitchell died in 2019 from the ultra-rare disease that was ultimately named after him, Mitchell Syndrome. Michele and her family started the Mitchell & Friends Foundation to support families and raise both awareness and money for research into Mitchell Syndrome. There have been over 25 patients identified worldwide since 2019. Michele lives in St. Louis, MO and is currently working to complete her Doctorate in Nursing Practice at the University of Missouri-Columbia.

Bret Koncak

Bret Koncak, Co-Founder, mejo

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 nurses and strategic administrators providing him with a comprehensive understanding of the healthcare landscape. Yet, it was his personal journey as a caregiver, navigating the challenges of his two sons’ sudden and severe rare diseases, that truly transformed his perspective. This firsthand experience highlighted the gaps in tools purpose-built for parents and caregivers like him, leading him to co-found mejo alongside his co-founder and fellow rare disease dad, Ryan Sheedy.

Bret lives in Prairie Village, KS with his wife Julee, and their children, Josie, Logan, & Camden.

Bryan Moore

Dr. Bryan Moore, Ph.D, Medical Affairs Executive Director Genentech

Bryan Moore is currently the Medical Affairs Executive Director at Genentech and oversees the medical and research strategy across multiple disease areas including rare diseases for the Midwest. Previously he has served in leadership roles at Roche Diagnostics, Chemicon/Merck, and other companies and has over 25 years of healthcare experience. His passion for rare diseases is driven by his scientific curiosity but also from his own child’s journey to be properly diagnosed and find treatment for a rare disease.

Josh Moore

Josh Moore,Director of Pharmacy for MO HealthNet Division (Missouri Medicaid)

Josh graduated from St. Louis College of Pharmacy in 2009 and joined MO HealthNet in 2019. As the Director of Pharmacy for MO HealthNet, Josh oversees the clinical management for medications, including developing prior authorization criteria and drug reimbursement methodologies. He also oversees the preferred drug list program, rare disease medications, and several advisory committees.

A married father of two daughters, and an avid runner, Josh has practiced pharmacy in the community setting and as a clinical pharmacist. He also has years of experience managing complex healthcare IT environments. With this background his goal is to enable and support access to the best treatment for patients in a fiscally responsible and sustainable way.

Doug Myers

Doug D. Myers, MD, Physician and Section Chief of Bone Marrow Transplantation, Children's Mercy Hospital, Professor of Pediatrics, University of Missouri-Kansas City School of Medicine; Clinical Associate Professor of Internal Medicine, University of Kansas School of Medicine

All creatures have a spectacular arrangement of cells. When these cells are at their best, the body functions properly. The opposite is true, however, when these cells decline. For Dr. Doug Myers, Physician and Section Chief of Bone Marrow Transplantation at Children’s Mercy, his objective is to assist and support families when this cellular misfire affects children with cancer. Myers focuses on modifying cell treatments and chimeric antigen receptor (CAR) T-cells to target pediatric cancer.
Myers’s motivation is simple and profound, he has seen too many parents leave the hospital without their kids. This sense of urgency has guided his career and work focused on clinical trials of cell therapies targeting cancer. Prior to moving to Kansas City, Myers worked in Houston on one of the first human trials of CAR T-cells. That involvement expanded upon relocating, as the Kansas City region provided an excellent collaborative opportunity to partner with local laboratories to develop cell therapies. “The CAR T-cell therapies are kind of like an investment portfolio, you need to diversify the types you use because certain strategies may not work as well as you think,” Myers said.

The CAR T-cells were originally developed in the late 1980’s but it wasn’t until recently that they were commercialized. For Myers, his role is understanding how CAR T-cells work to kill cancer, expand on that, and find novel ways to make them more effective. “We build on the work of others. If we can make the CAR T-cells a little more aggressive when they arrive at a tumor, we can get them reinvigorated and they will target the cancer,” Myers said.

By using technology that is already commercialized, it provides an opportunity for Myers to make an immediate translational difference. His laboratory at Children’s Mercy moves targeted therapies from bench to bedside to affect a wide array of patients. “Through recruiting and partnership, we see an opportunity to work in conjunction with an immunotherapy center that would expand the treatment to patients with solid organ transplant tolerance, autoimmune disease, things like that,” Myers said. With this focal point, he is able to better collaborate with the University of Kansas and other institutions to expand the research footprint in the region.

Myers acknowledges his success at Children’s Mercy is a result of the atmosphere that they have built and provided. “I see the need to collaborate and that’s more fun than trying to accomplish things on your own. There’s definitely a feeling of teamwork here and that goes a long way to being satisfied with your job,” Myers said. “We’ve built collaborations involving things as divergent as a cardiology lab at Children’s Mercy helping the T-cell lab at the University of Kansas Medical Centerto help both adults, children, and the animals at Kansas State University by collaborating with them through some of their animal models.”

This type of immunotherapy research may focus on pediatric cancer, yet its benefits can be seen with targeting cancer in adults and even animals. One Health is a collaborative approach of leveraging the linkage between human health, animal health, and the environment – a unique strength in the Kansas City region. “If we are doing animal testing as a necessary part of developing human treatments, why wouldn’t we get the benefit to the animals as well,” Myers said.

Myers’ interest in science emerged in his 9thgrade biology class when his teacher encouraged the class to imagine and draw the appearance of a virus. Myers was captivated in that moment when the teacher explained what a virus did and how they attacked the body.

There is also a foundation of support and hope of positive outcomes that Myers appreciates. He recalls an allogeneic stem cell transplant that carried a high price tag for the hospital but stood a good chance of helping a pediatric cancer patient. “I saw them coming from a distance, and I thought, oh no. They are going to tell me I can’t do this; we can’t take this risk” Myers said. Instead, they listened to the science, heard the potential impact it could have for this child, and committed to making that difference “I knew from that moment on that I was going to be supported,” Myers said.

Beyond cancer and illness, the motivation for Myers is to improve lives. His work has the ability to have an immediate impact and with strong collaboration, he will likely see more kids going home with their parents.

Dr. Tomi Pastinen

Dr. Tomi Pastinen, MD, PhD, Director of the Genomic Medicine Center (GMC) at Children’s Mercy Kansas City

Dr. Tomi Pastinen has been the Director of the Genomic Medicine Center at Children’s Mercy Kansas City (CMKC) since 2017. Dr. Pastinen joined Children’s Mercy from McGill University where he co-led the McGill Genome Center and supervised a multidisciplinary human genomics team 2006-2017 as a Professor of Human Genetics. He held a Canada Research Chair from 2007 to 2017 and has authored over 200 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, which produced maps of human genome function to the global research community. Dr. Pastinen earned MD PhD from University of Helsinki, Finland followed by post-doctoral training at McGill University and was mentored by Drs. Leena Peltonen and Thomas J. Hudson, respectively. Currently he is leading Genomic Answers for Kids, a foundational initiative shaping genomic medicine at Children’s Mercy that applies the most advanced genome testing platform to thousands of patients and their family members to solve unsolved rare diseases in children.

Nancy Petersen

Nancy Petersen, Community Liaison, Ability KC

Nancy Petersen received a MSW from Jane Addams School of Social Work at UIC. She currently works as a Community Liaison for Ability KC, a medical rehabilitation program in Kansas City, Missouri. She has spoken at many conferences and has contributed to two college level textbooks. She’s a member of Children’s Mercy Hospital Rare PFAC, serves as a parent advisor on the Children’s Mercy Research Institute Genomic Answers 4 Kids ELSI Committee. She serves on the advisory group for the Rare Voices Program-PCORI grant and is a member of the Rare Disease DEI Steering Committee Global Genes Project. She also sits on many conference planning boards in the KC area.

Nancy has been married 23 years to Jimmy, has a daughter who is a junior at Missouri State and a rare son who is a senior in high school. Oh, and a crazy service dog, Dwight, and two 13 yr old cats.

Kelly Ranallo - RareKC Founder/Governance

Kelly Ranallo, Founder RareKC and Founder/President of the Turner Syndrome Global Alliance (TSGA)

Kelly Ranallo is the founder of RareKC and Founder/President of the Turner Syndrome Global Alliance (TSGA). 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

Dennis Ridenour, President & CEO, BioNexus KC

Dennis Ridenour is President and Chief Executive Officer of BioNexus KC, the coordinating body for the KC region’s life sciences research initiatives. In this role, Mr. Ridenour fosters regional collaboration while working to increase the concentration and capacity for life sciences and healthcare research in the region. His work makes the region more efficient at commercializing technologies being developed locally, improves the health of patients and animals across the region while reducing and eliminating health disparities, and positively impacts the regional economy by leveraging our strengths at the intersection of human and animal health. Mr. Ridenour is strongly committed to the vision of BioNexus KC, that Kansas City is a global leader at the nexus of human and animal health benefiting all our citizens and the economy.

Prior to joining BioNexus KC, Mr. Ridenour was President and Chief Executive Officer of BioKansas, a regional life science and healthcare trade association. There he managed all facets of the organization, including day-to-day operations, member development and engagement, strategic planning, and finance. Mr. Ridenour previously worked at the Stowers Institute for Medical Research in Kansas City, MO, Merck Research Labs in Boston, MA, and as a researcher in the Department of Neurobiology at Harvard Medical School. Dennis earned a B.S. in Biological Sciences and an M.S. in Veterinary & Biomedical Sciences from the University of Nebraska-Lincoln, as well as an M.B.A. from the University of Kansas.
Mr. Ridenour serves as Chair of the Board of Directors for RareKC, a non-profit whose mission is to accelerate the diagnosis, care and treatment of rare conditions, and as a member of the Board of Directors for the Alphapointe Foundation, a non-profit that works to empower people with vision loss to live, work and be independent. He also currently serves on several other advisory boards whose work is committed to workforce development and the fight to eliminate health inequities and the systemic barriers that cause them.

Dr. Brett W. Sperry

Dr. Brett W. Sperry, Cardiologist, Saint Luke's Mid America Heart Institute, Associate Professor of Medicine, the University of Missouri-Kansas City

Dr. Brett W. Sperry is a cardiologist at Saint Luke’s Mid America Heart Institute with a subspecialty in Advanced Heart Failure, Transplantation, and Mechanical Circulatory Support. He is an Associate Professor of Medicine at the University of Missouri-Kansas City. Dr. Sperry was born in Connecticut and completed an undergraduate degree in Economics followed by medical school and Internal Medicine residency at Georgetown University in Washington, DC. He completed fellowship training in both Cardiovascular Medicine and Advanced Heart Failure and Transplantation at the Cleveland Clinic in Cleveland, Ohio.
He is board certified in Internal Medicine, Cardiovascular Medicine, Cardiovascular CT, Advanced Heart Failure and Transplantation, Nuclear Cardiology, and Echocardiography. His clinical and research interests include heart transplantation, mechanical circulatory support, cardiomyopathies (such as cardiac amyloidosis, sarcoidosis, and hypertrophic cardiomyopathy), and nuclear imaging.

Joshua Staley

Joshua Staley, Doctoral student, the University of Missouri-Kansas City School of Medicine

Joshua Staley is a skilled data scientist and researcher with a keen focus on genomics, animal health, and clinical human research. He earned his Bachelor of Science degree in Biology from the University of Missouri-Kansas City, followed by a Master of Science degree in Veterinary Biomedical Sciences from Kansas State University. Currently, Joshua is advancing his academic journey as a doctoral student at the University of Missouri-Kansas City School of Medicine. At the University of Kansas Medical Center, where he serves as a Senior Research Associate, studying triple negative breast cancer. His diligent research efforts have led to the publication of several abstracts, posters, and manuscripts.
Joshua stands out for his practical and strategic approach to tackling complex problems, demonstrating his leadership skills in his field. His quick learning ability and self-starting nature continuously push him to develop and adapt in his profession.


Zita Surprenant, MD, MPH

Zita Surprenant, MD, MPH, is a retired emergency medicine and public health physician. Dr. Surprenant’s first grandchild, Kennedy, was born with Vici Syndrome, an ultra rare devastating neurodevelopmental and neurodegenerative multi-system disease which is uniformly fatal. Since Kenney’s diagnosis, she has been a dedicated advocate for the Vici community, serving to advance research here in the US and Europe. Additionally, she provides families with support, resources and much needed understanding and love, as they navigate this terrible disease.

Jay Vivian, PhD

Dr. Jay Vivian, PhD, Director, Rare Disease Model Research Program, Associate Professor of Pediatrics, University of Missouri-Kansas City School of Medicine; Research Associate Professor of Pediatrics, University of Kansas School of Medicine

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.

Dr. Scott J. Weir, Pharm.D. Ph.D.

Dr. Scott J. Weir, PharmD, PhD, Director, Institute for Advancing Medical Innovation (IAMI), University of Kansas Medical Center

Scott J. Weir 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, 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. In 2015, IAMI established a preferred partnership with BioNovus Innovations LLC, providing a clear path to commercialization of medical innovations. This public-private partnership has advanced to a unique product development-focused translational research model CureBridgeCollaborative with six Kansas City area biotechnology companies formed to date.

Weir has over 37 years of professional experience in the field of drug discovery and development, 20 of which were spent in the pharmaceutical industry prior to joining KUMC in 2006. During his tenure in the pharmaceutical industry, Weir contributed to the development and registration of over 26 drug products. He is co-inventor of fosciclopirox, a drug treatment for bladder cancer and acute myeloid leukemia that has advanced to early phase clinical trials in cancer patients. His specific areas of expertise are in clinical pharmacology and developing innovative approaches to drug discovery and development. He has built a reputation of being innovative in bridging the “Valley of Death” through high performance public-private partnerships as well as repurposing FDA-approved drugs and rescuing abandoned drugs. Such partnerships have resulted in the creation of a robust cancer drug discovery portfolio of approximately 30 projects as well as advancement of five KU-invented anticancer agents to clinical trials over the past six years. Epaned™, developed in partnership with Silvergate Pharmaceuticals Inc. and Children’s Mercy Kansas City, was FDA-approved and launched in 2013.

From 2012-2017, Weir served on the National Center for Advancing Translational Sciences (NCATS) Advisory Council as well as the Cures Acceleration Network (CAN) Board. He co-chaired the NCATS Working Group that developed goals and objectives for the NIH Clinical and Translational Science Award program. Weir also participated on the Institute of Medicine Mapping the Drug Discovery and Development initiative and served on the NCI STTR/SBIR Working Group, advising this small business granting program on initiatives designed to strengthen collaborations with NCI-designated cancer centers. Weir served on the Patient Engagement Working Group within the Clinical Trial Transformation Initiative (CTTI) that released its recommendations on best practices in engaging patients in the research process and currently serves on the CTTI Steering Committee. Weir serves on scientific and external advisory boards for Family Health International 360, the Myeloproliferative Neoplasm Research Foundation, Center for Clinical and Translational Science at the University of Kentucky, and Midwest Stem Cell Therapy Center. He also serves on the Board of Directors for the Leo and Anne Albert Institute for Bladder Cancer Care and Research as well as in an advisory capacity to several biotechnology and pharmaceutical companies.

Melissa Wirtz

Melissa Wirtz M.A. CCC-SLP, Speech-Language pathologist

Melissa Wirtz is the mother of 2 young kids Colette (7); George (5), who has multiple rare diagnoses. Melissa is a Speech-Language pathologist with 10 years of clinical experience working in the early childhood educational setting with children with a wide variety of disabilities. She is also a member of the Rare PFAC at Children’s Mercy and serves in the Rare Voices program. She enjoys spending time with her kids, husband Mike; their dog Hattie as well as reading, cooking & walking.