Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4
Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4 1024 783 Abbie Miller

Recently published in Nature Medicine, interim results from a Phase 1/2 trial offer promising results.   Limb-girdle muscular dystrophy (LGMD) 2E/R4 is a rare, progressive neuromuscular disorder caused by mutations in the β-sarcoglycan (SGCB) gene. These mutations lead to SGCB protein deficiency, ultimately resulting…

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Dr. Jayanthi standing in OR
When Surgery Does Not Go as Planned
When Surgery Does Not Go as Planned 1024 575 V. Rama Jayanthi, MD

A surgeon’s reflections on complications, trust and moving forward together.   I have been thinking back on my career and the instances where a patient developed a complication, and the surgery did not go as planned. Having a family state their child was better…

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Dr. Iobst with patient
Magnetically Controlled Limb Lengthening Devices Safe for Patients With Programmable Implantable Devices, Study Shows
Magnetically Controlled Limb Lengthening Devices Safe for Patients With Programmable Implantable Devices, Study Shows 1024 683 Erin Gregory

Recent study evaluated the safety and efficacy of using magnetically controlled intramedullary nails in patients with programmable implantable devices, shedding light on a previously unexplored area of orthopedic surgery. In a pivotal study published in the National Center for Biotechnology Information, led by Christopher…

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Addressing Gastrointestinal Disorders to Improve Quality of Life for Individuals With Down Syndrome
Addressing Gastrointestinal Disorders to Improve Quality of Life for Individuals With Down Syndrome 1024 661 Erin Gregory

Down syndrome (DS) affects approximately 1 in 700 children born in the United States, presenting with intellectual disability and distinct physical features. Alongside these challenges, individuals with DS often contend with various medical issues that impact their quality of life (QoL), including gastrointestinal (GI)…

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Stem Cell Study Opens Door to Understanding Development of Rare Form of Congenital Heart Disease
Stem Cell Study Opens Door to Understanding Development of Rare Form of Congenital Heart Disease 150 150 Pam Georgiana

Researchers use induced pluripotent stem cell technology and single-cell genomics to pinpoint abnormal cell development in hypoplastic right heart syndrome. A rare form of hypoplastic right heart syndrome (HRHS), pulmonary atresia with intact ventricular septum (PA-IVS), occurs when the structures on the right side…

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Higher Respiratory Severity Scores Associated with Bronchopulmonary Dysplasia-Associated Pulmonary Hypertension Risk
Higher Respiratory Severity Scores Associated with Bronchopulmonary Dysplasia-Associated Pulmonary Hypertension Risk 1024 683 Mary Bates, PhD

The RSS score is noninvasive and may be clinically useful for risk stratification in extremely preterm infants. Researchers at Nationwide Children’s report that elevations in the respiratory severity score were correlated with an increased risk of bronchopulmonary dysplasia-associated pulmonary hypertension in preterm infants. This…

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Adolescents With Concussion May Benefit from More Cognitive Activity as Early as the First Week After Injury
Adolescents With Concussion May Benefit from More Cognitive Activity as Early as the First Week After Injury 1024 575 Laura Dattner

A new study shows limiting screen time and returning to school early following a concussion may hasten recovery timelines. In a study published in British Journal of Sports Medicine, researchers with the Center for Injury Research and Policy, Division of Sports Medicine, Center for Biobehavioral…

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Introducing Nationwide Children’s Hospital’s Behavioral Health Learning Library
Introducing Nationwide Children’s Hospital’s Behavioral Health Learning Library 1024 683 Pam Georgiana

Access the free educational resource for health care providers interested in evidence-based courses. Jennifer B. Reese, PsyD, is a clinical child psychologist at Nationwide Children’s and manages behavioral health and training at Big Lots Behavioral Health Services. She is concerned about the national shortage…

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Gene Therapy for the Masses?
Gene Therapy for the Masses? 1024 683 Katie Brind'Amour, PhD, MS, CHES

Long-lived financial and logistical hurdles make bringing new gene therapy products to market a major challenge. To help bring more of these medical miracles to fruition, experts across industry, regulatory review, science and medicine have begun to problem solve together.  With the approval of…

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Neurorehabilitation for Children With Batten Disease
Neurorehabilitation for Children With Batten Disease 1024 683 Lauren Dembeck

Therapy should be tailored to each child’s needs. Batten disease comprises a rare group of related genetic disorders characterized by progressive neurodegeneration. The disease primarily presents in childhood as seizures, vision loss, and developmental regression. Neurorehabilitation services, including physical therapy (PT), occupational therapy (OT),…

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Hear Me Read: Novel Technology Approach to Reading Skills for Children with Impaired Hearing
Hear Me Read: Novel Technology Approach to Reading Skills for Children with Impaired Hearing 1024 683 Madison Storm

For children who are deaf or have impaired hearing, access to sound through tools like hearing aids, cochlear implants and/or intensive speech therapy is critical for developing speech and literacy. “The challenges faced are not the same as those an adult faces,” says Prashant…

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Meet Daphne©, a ChatBot for Pediatric Health Care
Meet Daphne©, a ChatBot for Pediatric Health Care 150 150 Madison Storm

Machine learning and artificial intelligence (AI)have exploded across the worlds of marketing and commerce in recent years. At Nationwide Children’s, clinicians and researchers are focusing on how AI and related technologies can be used to reduce clinician workloads and improve patient outcomes. Emre Sezgin,…

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Innovations in Pediatric Cancer and Transplants: Q&A With Sara K. Rasmussen, MD, PhD
Innovations in Pediatric Cancer and Transplants: Q&A With Sara K. Rasmussen, MD, PhD 150 150 Pam Georgiana

Sara K. Rasmussen, MD, PhD, is a transplant and pediatric surgeon in the Department of Abdominal Transplant and Hepatopancreatobiliary Surgery and an associate principal investigator in the Center for Clinical and Translational Research in the Abigail Wexner Research Institute at Nationwide Children’s Hospital. She…

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RSV in Infants and High-Risk Children: A Commentary on the Roll-out of a New Preventive Medication
RSV in Infants and High-Risk Children: A Commentary on the Roll-out of a New Preventive Medication 1024 575 Pam Georgiana

According to the Centers for Disease Control and Prevention (CDC), respiratory syncytial virus (RSV) causes more than 2 million outpatient visits, as many as 80,000 hospitalizations, and 100 to 300 deaths in children under 5 years old annually. Infants under 6 months of age…

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Teen girl with backpack
National Health Crisis: Treating Tobacco Dependence in Adolescents
National Health Crisis: Treating Tobacco Dependence in Adolescents 1024 575 Pam Georgiana

Partners For Kids® offers a free tool to support primary care providers use nicotine replacement therapy to increase smoking cessation success. In 2023, the Centers for Disease Control and Prevention (CDC) reported that more than 6.6% of middle school students and about 12.6% of…

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Featured Researcher – Rolf Stottmann, PhD
Featured Researcher – Rolf Stottmann, PhD 150 150 Alaina Doklovic

Rolf Stottmann, PhD, is a principal investigator in the Institute for Genomic Medicine at Nationwide Children’s Hospital and professor at The Ohio State University College of Medicine. He joined Nationwide Children’s in 2021. Dr. Stottmann has an unwavering interest in the genetics of structural…

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Acute Kidney Injury After Comprehensive Stage Two Palliation in Infants With Hypoplastic Left Heart Syndrome
Acute Kidney Injury After Comprehensive Stage Two Palliation in Infants With Hypoplastic Left Heart Syndrome 150 150 JoAnna Pendergrass, DVM

Infants with hypoplastic left heart syndrome who undergo the Hybrid pathway palliation commonly develop acute kidney injury after the comprehensive stage 2 procedure and may require advanced medical therapy.   In a recent Cardiology in the Young publication, Andrew Yates, MD, associate medical director…

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Innovations in Pediatric Nephrology and Urology: Spotlight on Congenital Urinary Tract Obstruction
Innovations in Pediatric Nephrology and Urology: Spotlight on Congenital Urinary Tract Obstruction 150 150 Erin Gregory

Congenital urinary tract obstruction poses significant challenges for pediatric patients, compelling innovative approaches in urology. Nationwide Children’s Hospital pioneers research in this field, focusing on understanding the complexities of urinary tract conditions. Two labs within the Kidney and Urinary Tract Center at Nationwide Children’s…

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Legislation Associated With Reduction in Amount of Opioids Prescribed at Discharge
Legislation Associated With Reduction in Amount of Opioids Prescribed at Discharge 1024 681 Laura Dattner

Study shows a decrease after the 2017 Ohio Prescription Opioid Cap Law went into effect. The Ohio Opioid Cap Law, enacted on August 31, 2017, limited the total dose and duration of opioids that could be prescribed for minors. The anticipated benefit of cap laws…

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Novel Treatment Ameliorates Proteinuria and Dyslipidemia in a Model of Noninflammatory Glomerular Disease
Novel Treatment Ameliorates Proteinuria and Dyslipidemia in a Model of Noninflammatory Glomerular Disease 150 150 Lauren Dembeck

Idiopathic nephrotic syndrome — characterized by proteinuria, hypoalbuminemia, dyslipidemia, and edema — is one of the most common pediatric glomerular diseases. Glucocorticoids are first-line treatment, followed by calcineurin inhibitors for steroid-resistant disease; however, currently available calcineurin inhibitors can have numerous side effects, including dyslipidemia,…

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