Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease
Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease 1024 576 Abbie Miller

Researchers at Nationwide Children’s Hospital recently administered a novel gene therapy targeting SLC6A1, advancing precision medicine for children who previously could not be treated. Maxwell, age 8, made history this fall, when he received a bespoke gene therapy targeting his ultrarare disease. This wasn’t…

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3 Things to Know About Glomerular Disease
3 Things to Know About Glomerular Disease 1024 683 Gina Vitale, PTA

Gabriel Cara Fuentes, MD, PhD, offers insights for physicians regarding pediatric glomerular disease treatment and referral. Gabriel Cara Fuentes, MD, PhD, a pediatric nephrologist and principal investigator at the Kidney and Urinary Tract Research Center at Nationwide Children’s Hospital, is focused on a translational…

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Expanding the Evidence for Cannabidiol in Focal Epilepsy
Expanding the Evidence for Cannabidiol in Focal Epilepsy 1024 575 Pam Georgiana

A multicenter study led by Nationwide Children’s Hospital demonstrates long-term efficacy and safety of Epidiolex® for treatment-resistant seizures. As global use of cannabidiol for seizure management increases, most research has targeted generalized epilepsy. This leaves a major evidence gap for patients with focal seizures…

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As Suicide Increases in Preteens, Investigators Expand Research into Causes and Prevention
As Suicide Increases in Preteens, Investigators Expand Research into Causes and Prevention 1024 576 Katie Brind'Amour, PhD, MS, CHES

By tracking trends in suicide over time, researchers identify critical new directions for suicide prevention work. Last year, a study published in JAMA Network Open by researchers at Nationwide Children’s Hospital and collaborating institutions reported that suicide among preteens (children ages 8-12) had increased…

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Bringing Behavioral Health Into the Medical Home
Bringing Behavioral Health Into the Medical Home 1024 683 Pam Georgiana

Scaling integrated care across community pediatrics increases access to and utilization of mental health care. When a teenage patient shared feelings of anxiety and depression during a routine wellness visit, the pediatrician immediately called in the behavioral health clinician down the hall. Within minutes,…

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Featured Researcher – Sriram Vaidyanathan, PhD
Featured Researcher – Sriram Vaidyanathan, PhD 150 150 Gina Vitale, PTA

Sriram Vaidyanathan, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and an assistant professor of Pediatrics at The Ohio State University College of Medicine. Dr. Vaidyanathan’s path pivoted…

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Rethinking Neonatal HSV Management: A Less Aggressive, Evidence-Informed Approach
Rethinking Neonatal HSV Management: A Less Aggressive, Evidence-Informed Approach 1024 593 Alaina Doklovic

An alternative approach to neonatal HSV management helps keep mom and baby together during the nursery stay.  Neonatal herpes simplex virus infection (HSV) is a rare but often fatal disease when not treated correctly or in a timely manner. This makes diagnosis and preventive strategies extremely important when expectant mothers have active genital HSV infection when they deliver.   Neonatal HSV…

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Kids With GPA Often Show Lung Signs — But Few See Pulmonologists
Kids With GPA Often Show Lung Signs — But Few See Pulmonologists 1024 683 Jessica Nye, PhD

Most patients with pediatric granulomatosis with polyangiitis (GPA) have respiratory manifestations, but fewer than half are evaluated by a pulmonologist within 6 months of diagnosis, finds a study published in Pediatric Pulmonology.   “Pediatric GPA is a very rare autoimmune rheumatologic disorder that can have…

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Prematurity Increases Morbidity and Mortality Risk in Preterm Neonates with Down Syndrome in the NICU
Prematurity Increases Morbidity and Mortality Risk in Preterm Neonates with Down Syndrome in the NICU 1024 624 JoAnna Pendergrass, DVM

  Premature neonates with Down syndrome who are admitted to the neonatal intensive care unit experience higher rates of health complications and an increased mortality risk.   A retrospective study led by Emily Messick, DO, at Nationwide Children’s Hospital, reported the increased risk of…

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Molly Fuchs, MD
Rethinking Routine Kidney Ultrasounds in Children With Mild Anorectal Malformations
Rethinking Routine Kidney Ultrasounds in Children With Mild Anorectal Malformations 1024 683 Lauren Dembeck

  Annual scans rarely identify new kidney concerns in symptom-free children. Children born with anorectal malformations (ARMs), a group of congenital conditions affecting the anus and rectum, are routinely screened for kidney abnormalities because of the association between ARM and urinary tract anomalies. The…

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Featured Researcher: Diana Zepeda-Orozco, MD
Featured Researcher: Diana Zepeda-Orozco, MD 150 150 Gina Vitale, PTA

Diana Zepeda-Orozco, MD, is a principal investigator in the Kidney and Urinary Tract Research Center within the Abigail Wexner Research Institute, an attending physician and the associate division chief of research for the Division of Pediatric Nephrology and Hypertension, and an associate professor of…

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Challenges in Medication Titration in Children with Heart Failure
Challenges in Medication Titration in Children with Heart Failure 1024 537 Pam Georgiana

Beyond the guidelines: implementing consensus-based care for complex patients. Pediatric heart failure is a complex clinical diagnosis, with a growing body of evidence to suggest there are critical differences between pediatric and adult heart failure. In a recent review published in JHLT Open, Jessie…

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Defining Urinary Tract Infection: Why Standardization Matters for Research and Care
Defining Urinary Tract Infection: Why Standardization Matters for Research and Care 1024 683 Pam Georgiana

Urinary tract infections (UTIs) are one of the most common bacterial infections worldwide, affecting an estimated 150 million people annually and costing more than $5 billion in the United States alone. However, despite their prevalence, there is still no universally accepted definition of what…

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What Clinicians Need to Know About RSV Prevention
What Clinicians Need to Know About RSV Prevention 1024 609 Jason Newland, MD, MEd

Respiratory syncytial virus (RSV) infects the nose, throat and lungs, causing mild cold-like symptoms (upper respiratory infection) for most children and adults. However, in high-risk populations, including infants, children with chronic conditions, and older adults, it can be quite severe — ultimately leading to…

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Nationwide Children’s Team Builds Award-Winning AI Tool to Drive Mental Health Research
Nationwide Children’s Team Builds Award-Winning AI Tool to Drive Mental Health Research 1024 502 Abbie Miller

DREAM project wins the 2025 Gartner Eye on Innovation Mid-Size Enterprise Award in the Americas region. In recognition of its innovative application of technology to address critical health care challenges, the Office of Data Sciences was selected as the winner for the prestigious 2025…

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Food-Based Tube Feeding: Balancing Parental Demand With Pediatric Nutrition Science
Food-Based Tube Feeding: Balancing Parental Demand With Pediatric Nutrition Science 1024 575 Pam Georgiana

New research from Nationwide Children’s reveals significant differences in nutrient adequacy across commercial food-based formulas. In the last two decades, enteral nutrition has undergone a significant transformation. Fifteen to 20 years ago, pediatric patients requiring gastrostomy tube feeding had only one option: synthetic, cow…

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Guiding the Treatment of Ballistic Fractures: A New Report Offers Education and Support for Orthopedic Surgeons
Guiding the Treatment of Ballistic Fractures: A New Report Offers Education and Support for Orthopedic Surgeons 1024 620 Alaina Doklovic

Despite firearm injury incidence and severity in children, no guidelines for orthopedic surgeons existed – until now. Firearm-related injury is the leading cause of death in the United States among children and adolescents aged 1 to 19. Despite this alarming statistic, there were no…

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Parents’ Perceptions Highlight Gaps in Supporting Children with Epilepsy in School
Parents’ Perceptions Highlight Gaps in Supporting Children with Epilepsy in School 1024 579 JoAnna Pendergrass, DVM

Discussions with parents of children with epilepsy revealed concerning gaps in school-based seizure education and care, underscoring the need for better lines of communication between medical teams, school personnel, and the children’s families.   A qualitative study led by Mary Kay Irwin, EdD, and…

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Closing the Treatment Gap: Opioid Use Disorder Medications in Adolescents and Young Adults
Closing the Treatment Gap: Opioid Use Disorder Medications in Adolescents and Young Adults 1024 683 Pam Georgiana

Findings from the PROUD trial highlight the need for youth-centered prescription strategies in primary care. Adolescents and young adults are far less likely than older adults to receive evidence-based medications for opioid use disorder (OUD), even though buprenorphine and extended-release naltrexone are recommended treatments.…

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Advances in the Care of Patients With Chest Wall Deformities
Advances in the Care of Patients With Chest Wall Deformities 1024 683 Sara Mansfield, MD

Every year Nationwide Children’s Hospital sees several hundred children and adolescents with chest wall deformities. Common Chest Wall Conditions Two of the most common chest wall diagnoses are pectus excavatum (sunken chest) and pectus carinatum (pigeon chest).  These conditions can cause a variety of…

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