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Transforming Pediatric Medicine Through Breakthroughs and Discovery
With more than 30 pediatric specialties and the strength of an academic medical center behind it, UHealth Jackson Children’s is redefining what advanced pediatric care looks like in Florida
| 8/3/2026
Years later, Pankaj Agrawal, M.D., still remembers saying those words to the parents of a 3-week-old infant who died in the newborn intensive care unit from an unknown condition. The devastated couple wanted answers
Dr. Agrawal, physician-in-chief and chief of neonatology at Holtz Children’s Hospital, the flagship of UHealth Jackson Children’s, kept his promise. Through his work in advanced genetic research and rare diseases, he continued investigating the infant’s case long after her death
Six months later, the neonatologist and renowned researcher and his team identified the baby’s underlying genetic condition. The discovery allowed the couple to pursue early genetic screenings in subsequent pregnancies. They now have three healthy children and have raised $1 million to support Dr. Agrawal’s research
For Dr. Agrawal, the story reflects the mission of academic pediatric medicine, combining discovery, innovation, and clinical expertise to impact patients’ lives. The National Institute of Health has funded his original research on genomics, rare diseases, and its applications in prenatal and neonatal care for more than two decades
“Parents deserve answers,” he said
That philosophy positions UHealth Jackson Children’s as a pediatric superpower in Florida. A place where doctors care for children today, while shaping the future of medicine through groundbreaking research and training the next generation of doctors in a pediatric residency program ranked number one in Florida by Doximity Residency Navigator
Moving Medicine Forward
Rooted in academic medicine since 1952, UHealth Jackson Children’s is home to Florida’s first accredited and longest-standing academic pediatric program, bringing breakthrough discoveries directly to every child’s bedside
“We translate our knowledge into real benefits for our patients,” Dr. Agrawal said. “We are working firsthand on complex conditions, building new technologies, and developing new approaches. That is our unique advantage.”
Nowhere is that more evident than in Holtz Children’s work in advanced genetic research and rare disease diagnosis. While most hospitals rely solely on commercial laboratories for whole genome sequencing, Dr. Agrawal’s team goes a step further when such testing produces no answers
“What sets us apart is the research we do,” he said. “When clinicians say there is no diagnosis, that’s when we get to work.”
At the Rare Disease Center in UHealth Jackson Children’s, physician researchers use advanced genomic analysis and a growing database of more than 10,000 rare disease patients. The team reanalyzes genetic data to identify mutations, discover new genes, and develop precision therapies
Genetics is Only Part of the Story
Throughout the organization, multidisciplinary teams collaborate across specialties to solve some of the most complex pediatric cases in medicine
“We are uniquely qualified to handle complex cases where multiple organ systems are involved,” Dr. Agrawal said
At UHealth Jackson Children’s, complex care often begins before birth. The nationally recognized maternal-fetal medicine and fetal surgery teams coordinate highly specialized interventions for babies in utero. This includes advanced fetal interventions such as in utero vein of Galen surgery for rare and life-threatening neurological conditions. In some cases, more than a dozen specialists are assembled before delivery to prepare for immediate treatment after birth
Cross-discipline collaboration is key for developing personalized care plans in complex cases. For example, the comprehensive spina bifida clinic regularly provides coordinated treatment plans from pediatric specialists in neurosurgery, urology, orthopedics, rehabilitation, transplants, and more
Similarly, cardiology expertise spans fetal interventions, minimally invasive procedures, newborn and pediatric heart surgery, electrophysiology, ventricular assist devices, and hybrid procedures, combining catheter-based interventions with surgery to treat critically ill newborns
UHealth Jackson Children’s also provides continuity of care that standalone children’s hospitals often cannot offer. Children with congenital heart disease, sickle cell disease, or cystic fibrosis can transition seamlessly into adult specialty care within the same health system
Advancing the Next Generation of Pediatric Medicine
Pediatric medicine is evolving rapidly, particularly in areas such as genomics, regenerative medicine, and gene therapy
UHealth Jackson Children’s is already bringing many of these advances to patients through various clinical trials and early access to innovative therapies for conditions including sickle cell disease, Duchenne muscular dystrophy, and spinal muscular atrophy
Leadership sees an even bigger opportunity ahead: expanding pediatric clinical trials and translational research that can accelerate breakthroughs for children nationwide
“One of my goals is to make us a powerhouse for pediatric clinical trials,” Dr. Agrawal said. “We’re the right place for this work because we’re an academic center with a strong team. We have the ability to conduct research, pursue funding, and develop new treatments that can truly make a difference.”
Visit MiamiRareDiseaseCenter.org to learn more about our program. For more information on our services, visit UHealthJacksonChildrens.org
Sunshine Genetics Act
Florida’s Ambitious Screening Program is Nation’s First
A new pilot program to screen Florida newborns for more than 1,000 potentially treatable genetic conditions (only 60 conditions are screened currently) is a game changer, said Pankaj Agrawal, M.D., who is co-leading the genomic newborn screening program with geneticist David Ledbetter, Ph.D., at Florida State University. The Florida Legislature passed legislation to create the program, placing Florida at the forefront of newborn screening using advanced genomic sequencing for newborns
About UHealth Jackson Children’s
UHealth Jackson Children’s at Holtz Children’s Hospital is home to more than 30 pediatric specialties. As South Florida’s longest-standing academic children’s hospital, it combines advanced research, leading-edge treatments, and multidisciplinary expertise to care for some of the region’s most complex pediatric conditions. Nationally recognized programs include fetal medicine, cardiology, rare diseases and advanced genomics, transplant, orthopedics, oncology, and one of the nation’s premier Level IV newborn intensive care units.
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