Growing Academic Preeminence
How We Impact the Future
Through our multi-state, comprehensive health system, we have the unique opportunity to scale our impact across geographies while advancing and sharing knowledge through scholarly research and medical education.
Research in 2024
$81M
Total Research Funding
395
Funded Research Projects
305
Clinical Trials
594
Publications
Research Breakthroughs
Duchenne Muscular Dystrophy Gene Therapy
Omer Abdul Hamid, MD, is leading research on gene therapy for Duchenne Muscular Dystrophy, which has shown remarkable early success. Five patients have received the treatment so far and have demonstrated improvement within weeks of receiving the therapy, regaining physical capabilities that have led to unexpected outcomes due to increased activity. The treatment was approved through the FDA’s accelerated approval pathway. The therapy requires a two-month isolation period post-infusion to prevent infections that could compromise outcomes.
See Jaxson’s remarkable progress after receiving gene therapy.
The success of this Duchenne Muscular Dystrophy gene therapy builds on previous breakthroughs in spinal muscular atrophy treatment and holds promise for numerous other genetic conditions. While not a cure, it represents the most significant advancement in Duchenne Muscular Dystrophy treatment to date. The research team is now expanding to international patients, with our first non-U.S. patient expected soon.
“This innovative new gene therapy will treat the cause of the disease, giving a lot of hope to the Duchenne Muscular Dystrophy community, including doctors, researchers, patients, families, and advocates that have been searching for answers for a long time.”
— Omer Abdul Hamid, MD
Neuromuscular Neurologist, Nemours Children’s Hospital, Florida
Research Breakthroughs
Zika Virus Research Updates
Researchers Tamarah Westmoreland, MD, PhD, and Joseph Mazar, PhD, have made a significant breakthrough in their investigation of Zika virus as a treatment for neuroblastoma, a rare childhood cancer. Their study, published in Cancer Research Communications, demonstrated remarkable results in models where injections of Zika virus either dramatically shrank or completely eliminated neuroblastoma tumors.
While the researchers emphasize that extensive additional studies on safety and efficacy are still needed before clinical application, this innovative approach could potentially offer new hope for the more than half of high-risk neuroblastoma patients who don’t respond to conventional treatments.
Breakthrough Continues: In August, Pass It On To Kids Foundation announced a $7 million gift to accelerate the pioneering research using Zika virus to fight neuroblastoma and ovarian cancers.
2024 Research Highlights
Nelly Mauras, MD, NIH U01
5-year $2,400,000 grant
“Cognitive, Structural, and Functional Impact of New-Onset Type 1 Diabetes on the Brain of Young Children: Understanding Risks and Protective Factors.” This grant from the National Institute of Diabetes and Digestive and Kidney Diseases is a continuation of a long-standing line of work by Dr. Mauras and her team through several funding cycles studying the impact of type 1 diabetes on the brain in children.
Alexandra Monzon, PhD, NIH K23
5-year $754,176 grant
“Adapting Single Sessions Interventions for Type 1 Diabetes”: Integrated Pediatric Care to Reduce Depression and HbA1C” The proposed research is to improve the health and quality of life of individuals living with diabetes by using a novel single-session intervention approach to improve depressive symptoms and glycemic levels in youth with type 1 diabetes. This will result in an innovative type 1 diabetes-specific depression single-session intervention with the potential to significantly improve health and access to mental health care for adolescents with type 1 diabetes who report elevated depressive symptoms.
Sree Viswanathan, MD, NIH R01
3-year $546,480 grant
“Oral Feeding Difficulty in Large for Gestational Age Infants: Defining Interrelationships Between Body Composition, Oral Feeding Ability, and Appetite-Regulating Hormones”
This study will evaluate the role of body composition, appetite regulating hormones, and the implementation of a novel infant feeding strategy and its impact on length of hospital stay and short-term clinical outcomes in large-for-gestational age infants.
Amanda Hernan, PhD, NIH R01
5-year $2,000,000 grant
“Leveraging Genetically Encoded Heterogeneity to Understand TANDs and Seizures in Novel Models of TSC”
This study seeks to increase understanding of tuberous sclerosis complex (TSC), a leading genetic cause of epilepsy and autism spectrum disorder, in which noncancerous tumors compromise brain development and function. Up to 90% of patients diagnosed with TSC have epilepsy, which involves recurring seizures caused by abnormal electrical impulses in brain tissue.
Catherine Haut, DNP, and Aaron Carpenter, DNP, HRSA
4-year $2,400,000 grant
“Advanced Nursing Education – Nurse Practitioner Residency and Fellowship Program” This grant will provide pediatric primary care nurse practitioner fellows with extensive training in specialized behavioral and mental health care and other pediatric subspecialities. The grant helps increase the number of primary care nurse practitioners in community settings, including rural and underserved areas of Delaware and allows for the training of 16 fellows over the four-year period.
Next Gen Research
A Pipeline for New Investigators
Thao Ly Phan, MD, MPH, shares the importance of protected time, support, and mentorship she received through the Mentored Research Development Award Program.
In 2024, the Ginsburg Institute and the Center for Pediatric Clinical Research & Development announced the launch of the Mentored Research Development Award Program program in Florida. The program mirrors the model begun in Delaware in 2013 through an NIH-funded grant. The Mentored Research Development Award Program supports the development of competitive grant writing skills and helps early-career investigators make connections to potential collaborators at other institutions. To date, 35 of our physicians in the Delaware Valley have participated and benefited from the program.
In its 54th year, the Nemours Summer Undergraduate Research Program, funded by our Biomedical Research Department, welcomed 27 students across three sites. These students were selected from more than 200 applicants from 90 universities in 24 U.S. states. Our research scientists and clinicians serve as mentors and guide the scholars in a wide range of projects from sports medicine to cancer immunotherapy. In addition to mentor-directed research projects, the scholars participated in weekly enrichment seminars, a student-led journal club, an elevator-pitch competition, and an alumni career panel.
Medical Education in 2024
441
Residents
251
Fellows
456
Medical Students
2,668
Nursing Students
55
Nurse Residents
Partnership Highlights
The Magic of Collaboration
Academic partnerships, including collaborations with Mayo Clinic in Florida and the University of Florida in Jacksonville, the University of Central Florida in Orlando, and the University of Delaware and Thomas Jefferson University in the Delaware Valley, create a comprehensive approach to developing future pediatric health care leaders while advancing clinical excellence and research initiatives.
In April 2024, the University of Central Florida (UCF) College of Medicine – Nemours Children’s Health Department of Pediatrics was established. This expands our existing collaboration with UCF which provided training for more than 1,100 UCF medical students across various pediatric disciplines at Nemours Children’s Hospital, Florida, reaching all 480 UCF medical students annually.
The alliance strengthens joint commitments to pediatric medical training, workforce development and innovation while creating an institutional home for Nemours Children’s faculty. Beyond physician training, the partnership extends into interdisciplinary initiatives spanning athletics through the United States Tennis Association (USTA), hospitality workforce support, and technological innovations in engineering, optics, photonics and aerospace microgravity research. Additionally, in September, we announced a $5 million investment in pediatric nurse education, training, innovation and research at UCF’s Dr. Phillips Nursing Pavilion.
“Nemours Children’s shares our three missions. They are fully engaged in research, finding new cures and treatments. They provide care across the spectrum of pediatrics. And they are focused on education — of UCF medical students, residents and fellows. We are now expanding a great partnership that has allowed us both to thrive.”
— Deborah German, MD, Founding Dean, UCF College of Medicine, and Vice President for Health Affairs
Also in April, Mayo Clinic in Florida and Nemours Children’s Health, Jacksonville, announced the 10-year extension of a longstanding collaboration spanning three decades. The renewed agreement will expand educational programs for health care professionals, advance cooperative clinical and basic science research initiatives, and explore additional shared opportunities. The collaboration will enhance work to address significant shortages of pediatricians in Florida — 83 pediatricians per 100,000 children, below the national average — by furthering opportunities for Mayo Clinic residents, fellows and staff to receive essential specialty training and hands-on experience with our pediatric subspecialists.
“We are thrilled to continue our partnership with Nemours Children’s. Our unwavering commitment to improving patient care, conducting groundbreaking research and fostering education will undoubtedly have a positive impact on the future of health care.”
— Kent Thielen, MD, CEO, Mayo Clinic in Florida
The partnership has already been foundational to Mayo Clinic’s educational programs, including its medical school and numerous residency and fellowship programs. Our research efforts have benefited from access to equipment and technology available at Mayo Clinic. Additionally, collaborative clinical programs, such as stem cell transplant and CAR-T cell therapy, and research studies, such as type 1 diabetes impacts on developing brains, have flourished under the joint effort, to the benefit of families throughout Florida and around the world.