Dr. Andrea Gropman is an internationally recognized physician-scientist specializing in neurogenetics, rare disease, and neurodevelopmental disabilities. She served as the Division Chief of Neurodevelopmental Disabilities and Neurogenetics at Children’s National Hospital for 10 years, with a 25-year career at Children’s National Hospital where she rose from neurology fellow to a national leader and international rare disease expert and researcher before moving to St. Jude Children’s Research Hospital in 2024 to establish and direct a neurometabolic translational research program. Dr. Gropman also holds the position of Principal Investigator for the Urea Cycle Disorders Consortium. She is a tenured professor who held two endowed chairs: the Margaret O’Malley Chair in Genetic Medicine at CNH, and currently, the Mark F. Tamer Endowed Chair in Pediatric Neurology at St Jude.
Dr. Gropman is board-certified in Pediatrics, Neurology with Special Qualification in Child Neurology, Neurodevelopmental Disabilities, and both Clinical and Biochemical Genetics, reflecting her broad expertise in neurological and genetic disorders. She established one of the first neurogenetics clinics in the country in 1996 and the only neurogenetics division in 2013, which until her departure, had three dually trained (Neurology and Genetics) physicians.
Dr. Gropman completed college at Brandeis University. She majored in biology, where she got her first taste of science in the laboratory of Herman Epstein, Ph.D. a developmental biologist and physicist. She graduated from University of Massachusetts Medical School, where she continued to pursue neurology, this time in the Drachman Lab studying Alzheimer Disease pathology. She was awarded the Hewlitt Packard Top Graduate Award and The National Heart and Blood Prize in pathology. During medical school, she completed a rotation at Tufts with Dr. Paul Rosman, whom she credits with making the decision solidified to become a child neurologist. She also had the opportunity round with Dr. Raymond Adams. She trained at The Harriet Lane under Dr. Frank Oski. During her intern year, she “wandered” to the clinical research unit in the children’s unit, as it was known to house medical mysteries and rare disease. There, she saw her first patient with a urea cycle disorder being treated with a crude, over-the-counter drug that would later be approved as the first nitrogen scavenger. Little did she know, that now, 25 years later, she would spend her entire career in the pursuit of understanding the brain basis of these conditions and work to develop therapies for this and other rare diseases.
She thereafter trained in child neurology in the George Washington-Children’s National Hospital program. She was featured in the Washingtonian Magazine as “people to watch.” She then trained at the NIH National Human Genome Research Institute to complete fellowships in both clinical genetics and biochemical genetics. She met William Gahl, with whom she discussed unresolved cases for which she suggested there be a formal platform. Later, this became the Undiagnosed Disease Network. While at NIH she received The National Institutes of Health Award of Merit for “creating a landmark effort that brings hope to the hopeless.”
After her NIH training, Dr. Gropman took a job at Georgetown University, before returning to Children’s National. At Georgetown, she developed the CME course “Genetics for Your Practice,” recognizing the importance genetics would have across all specialties, and she received a grant from the National Coalition for Health Professional Education in Genetics to develop a genomics curriculum for neurologists, a course that was later acquired by the Jackson Lab for which she continues to review content.
Her research program really started at Georgetown. At the time, the congressional mandate for the first Rare Diseases Clinical Research Network intersected with her interest in imaging the brain in inborn errors of metabolism. Dr. Roger Packer, her chief, encouraged her to apply for a K12 award using imaging to study her longstanding interest in metabolism and the brain.
Dr. Gropman’s research has been instrumental in identifying how hyperammonemia affects brain metabolites and contributes to cognitive and neurological impairments. She explored how metabolic disruptions in UCDs cause structural and functional brain abnormalities, bridging the gap between metabolic dysfunction and clinical symptoms.
As the PI of the UCDC, she advanced clinical trials and natural history studies aimed at improving understanding, management, and treatment of UCDs. Her work has helped establish evidence-based guidelines for UCD management, including dietary and pharmacological interventions. She opened doors for young investigators to collaborate and be mentored by herself and the other faculty in the UCD consortium.
Dr. Gropman has made significant contributions to the field of neurology and genetics through her extensive involvement in educational and scientific program committees. She served on the CNS Annual Program Committee, Awards Committee, and Legislative Affairs Committees, as well as being the CNS liaison for the American Brain Coalition. She developed the Neurogenetics SIG, which started with three core members and has grown to over 100 attendees at the Annual Meeting. Dr. Gropman has leadership roles or major committee service within the ANA, the American Society of Human Genetics, the American College of Medical Genetics and Genomics, the Society for Inborn Errors of Metabolism, and the International Society for Magnetic Resonance in Medicine. Through her committee work, Dr. Gropman consistently championed multidisciplinary approaches, highlighting the importance of integrating genetics, neurology, and developmental medicine to advance patient care.
Dr. Gropman’s commitment to advancing education and scientific dialogue is evident in her dedication to selecting content that addresses current challenges and future directions in the field. Her work has helped position the society as a leader in providing meaningful, relevant, and impactful programming. These contributions reflect her passion for mentorship, lifelong learning, and advancing the field of neurogenetics, ultimately improving care for patients with complex neurological and genetic conditions.
Dr. Andrea Gropman is a prominent figure in patient advocacy, particularly within the realm of rare diseases. Her work in this area is multifaceted and includes:
Leading Research Consortia: She is the PI of the Urea Cycle Disorders Consortium and the UCDC Imaging Consortium, which are part of the Rare Disease Clinical Research Network. This work involves facilitating collaboration, study enrollment, and data sharing to advance medical research on rare diseases, and has led to the development of effective treatment strategies and government approval of life-saving therapies for urea cycle disorders.
Public Outreach and Policy Advocacy: Dr. Gropman actively engages in public discourse to promote the understanding and relevance of child neurology and neuroscience in society. She works with organizations like the National Urea Cycle Disorders Foundation, the United Mitochondrial Disease Foundation, and Parents and Researchers Interested in Smith-Magenis Syndrome.
Improving Access to Care: Dr. Gropman advocates for policies and practices that support collaborative, high-quality care for children with neurological disorders. She has participated in Hill activities on Rare Disease Day, and most recently, as an ambassador representing St. Jude to discuss the recent cuts in NIH funding and the impact it will have on rare disease families and therapies, and science.
Promoting Precision Medicine: She advocates for the involvement of child neurologists in precision medicine initiatives, ensuring they are at the table and equipped to order and interpret genetic tests and collaborate with genetic colleagues.
Mentorship: Dr. Gropman also finds reward in mentoring younger professionals, students, and newcomers to the field, supporting their growth and development and contributing to the future of child neurology. She won the 2023 Translational Mentor Award while at Children’s National Hospital.
Dr. Gropman has been married to Barry Gropman since 1990, and they have one daughter, Eliana, who was a former Team USA ice dancer and national and international medalist.
Publications and Research
Dr. Gropman is a prolific writer and has authored and co-authored 300 peer-reviewed articles, book chapters, and reviews focusing on the intersection of genetics, neurology, neuroimaging, and developmental disorders. She serves on NIH grants review committees. is an associate editor for the Journal of Child Neurology, section editor for Swaiman’s Pediatric Neurology, and special editor twice for the journal Neurotherapeutics, where she selected junior colleagues to showcase their work and provide visibility.
She has held NIH and other funding since 2005.
She is currently writing a book titled Looking for Zebras, and a screenplay, which explores her experiences as a physician working with families affected by rare diseases and the invaluable lessons she has learned from them, inspired by her desire to keep challenging herself, and her father’s influence as a journalist and community theater actor.
Legacy
Dr. Gropman’s career is marked by her dedication to improving the lives of patients with rare diseases and her role as a trailblazer in neurogenetics. Her work has left an enduring impact on the field, advancing both the science and the compassionate care of individuals and families affected by these challenging conditions.
Dr. Gropman is a master clinician known for her deep empathy, diagnostic acumen, and unwavering commitment to patients with rare and complex neurogenetic disorders. She is a consummate team scientist and trusted collaborator, leading interdisciplinary efforts that bridge clinical insight with cutting-edge research. An innovator in the use of advanced neuroimaging and systems biology to uncover biomarkers and therapeutic targets, she also brings a generous and inclusive spirit to mentorship. As a gracious sponsor, particularly of women and underrepresented trainees, she has championed the careers of countless junior colleagues. Tireless in her advocacy for patients and for the development of future leaders in medicine and science, Dr. Gropman models the highest ideals of academic medicine.