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Understanding the Social Neural Network
 Understanding the Social Neural Network 1024 575 Natalie Wilson
By: Natalie Wilson
Young boy turned sideways in front of a dark background, with dark lighting and an illustration of his brain appearing in front of the side of his head

Neuroimaging of participants with and without epilepsy allows researchers to explore the neural networks associated with social skills. Epilepsy is a chronic brain disease characterized by neural network dysfunction and seizures. According to the Centers for Disease Control (CDC), 470,000 children in the United States had active epilepsy in 2015. These children are more likely…

Bronchopulmonary Dysplasia: Characterizing the Chronic Lung Disease of Prematurity
 Bronchopulmonary Dysplasia: Characterizing the Chronic Lung Disease of Prematurity 1024 575 Lauren Dembeck
By: Lauren Dembeck
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

  Precise definitions and/or classifications of bronchopulmonary dysplasia are needed to optimize outcomes. Researchers at Nationwide Children’s Hospital are continuing to help refine the definition of severe bronchopulmonary dysplasia (sBPD). In a recent study, members of the BPD Collaborative demonstrated that preterm infants with sBPD who were on invasive mechanical ventilation at 36 weeks, a…

Diuretic Therapy in Preterm Infants With Acute Kidney Injury Is Common: Should It Be?
 Diuretic Therapy in Preterm Infants With Acute Kidney Injury Is Common: Should It Be? 1024 575 Lauren Dembeck
By: Lauren Dembeck
small baby with nose canula

Lack of efficacy and safety data on diuretic therapy in the critically ill infants is cause for concern. Researchers at Nationwide Children’s Hospital are beginning to fill in the gaps. Despite the common use of diuretic therapy in preterm infants with acute kidney injury, to date, no large studies have evaluated the patterns of prescription…

Chronic Pediatric Pancreatitis and (No) Persistent Belly Pain
 Chronic Pediatric Pancreatitis and (No) Persistent Belly Pain 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Underlying — even silent — chronic pancreatitis can be at play in children without previous acute pancreatitis episodes, including among children with little or no reported pain. Pancreatitis in children is rare (about 3-13 per 100,000 people per year), but diagnosis is increasing, at least in part due to increased awareness among physicians. The path…

Understanding Black Youth Suicide: Steps Toward Prevention
 Understanding Black Youth Suicide: Steps Toward Prevention 1024 596 Abbie Miller
By: Abbie Miller

To understand the how and why of rising rates of Black youth suicide, researchers call for a ‘Ground Zero’ approach. In December 2019, the Congressional Black Caucus released the statement “Ring the Alarm: The Crisis of Black Youth Suicide in America.” The statement focused on rising suicide rates among Black youth in the United States.…

“Left Behind Again:” Rural Children Enrolled in Medicaid Use Home Health Services Far Less Than Urban Children
 “Left Behind Again:” Rural Children Enrolled in Medicaid Use Home Health Services Far Less Than Urban Children 1024 575 Jeb Phillips
By: Jeb Phillips

Data from a pediatric accountable care organization is allowing physicians and researchers to explore the rural-urban health care gap. While it’s long been clear that children in rural areas have reduced access to some kinds of health care services compared to their urban peers, understanding the specifics of the care gap has proven difficult, because…

Study Highlights Relationship Between SI Gene and Chronic, Idiopathic Loose Stool in Some Children
 Study Highlights Relationship Between SI Gene and Chronic, Idiopathic Loose Stool in Some Children 1024 680 Abbie Miller
By: Abbie Miller
Close up color photo of little boy holding hands on his belly

Researchers conducted one of the largest prospective, multicenter trials in pediatrics looking at the connection between SI variants and common gastrointestinal symptoms. The SI gene codes for the sucrase-isomaltase enzyme, which is expressed in the intestinal brush border. This enzyme is important to the digestion of sucrose, and without it, maldigestion and poor absorption of…

Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network
 Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network 1024 683 Natalie Wilson
By: Natalie Wilson

The Children’s Oncology Group (COG), the world’s largest and most recognized pediatric cancer research collaborative, recently selected Nationwide Children’s Hospital to join its Pediatric Early Phase Clinical Trial Network (PEP-CTN) through a peer review process. The COG PEP-CTN is a re-iteration of the COG Phase 1/Pilot Consortium and is comprised of 21 core member sites…

Surgical Management of Recurrent Patellar Instability in the Pediatric Population
 Surgical Management of Recurrent Patellar Instability in the Pediatric Population 1024 575 Mary Bates, PhD
By: Mary Bates, PhD

Medial patellofemoral ligament reconstruction via the quadriceps turndown technique is safe and effective in young patients. In a new study, researchers from Nationwide Children’s Hospital demonstrate the safety and efficacy of the quadriceps turndown technique for medial patellofemoral ligament reconstruction in pediatric and adolescent patients. Patellar instability is a common knee condition in children and…

Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment
 Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment 1024 681 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
sleeping infant

A review of antibody titers for SMA patients revealed that levels may decrease with time, potentially enabling delayed dosing for children who otherwise might have been excluded from life-saving gene therapy. Adeno-associated viral (AAV) vectors are used to deliver gene therapies such as onasemnogene abeparvovec-xioi (ZOLGENSMA®), a recently approved gene therapy for spinal muscular atrophy…

Keeping in Touch: Renin-Producing Cells Require Cell Adhesion Molecule to Survive
 Keeping in Touch: Renin-Producing Cells Require Cell Adhesion Molecule to Survive 1024 575 Lauren Dembeck
By: Lauren Dembeck

Uncovering the molecular mechanisms underlying the renin-angiotensin-aldosterone system may provide insights into the long-term effects of hypertension and antihypertensive medications.  As the main source of renin in the human body, juxtaglomerular (JG) cells are crucial for blood pressure and fluid-electrolyte homeostasis. However, these cells cannot be cultured in vitro, making it challenging to study the…

The Sound of Silence: The Impact of “Silent” Genetic Variation in Health and Disease
 The Sound of Silence: The Impact of “Silent” Genetic Variation in Health and Disease 150 150 Lauren Dembeck
By: Lauren Dembeck

Despite widespread scientific interest in messenger RNA (mRNA) structure, its role in human health and disease remains poorly understood. Researchers at Nationwide Children’s Hospital have developed novel software and metrics to identify “silent” genetic variants that affect mRNA folding and may underlie rare genetic disorders. Historically, synonymous or “silent” genetic variants — those in protein-coding…

Pediatric Acute Care Cardiology Collaborative: Shared Focus on Quality to Improve Outcomes and Family Experiences
 Pediatric Acute Care Cardiology Collaborative: Shared Focus on Quality to Improve Outcomes and Family Experiences 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Illustration of heart, CAVD

The PAC3 has released three recent studies describing care practices and outcomes across member institutions in an effort to drive improved care. Established in 2014 with the aim of improving acute care outcomes and experience among pediatric cardiology patients, families, clinicians and hospitals, the Pediatric Acute Care Cardiology Collaborative (PAC3) has started to deliver on…

Unraveling Intractable Functional Constipation: Manometries Are Still the Gold Standard
 Unraveling Intractable Functional Constipation: Manometries Are Still the Gold Standard 1024 680 Natalie Wilson
By: Natalie Wilson
Close up color photo of little boy holding hands on his belly

Cine-MRI recordings can’t replace colonic manometries when assessing motility among children with functional constipation, but they may offer additional information. And conducting both MRIs and manometries simultaneously is feasible. While functional constipation (FC) is common in childhood, and most children who experience it respond well to behavioral interventions or treatment with laxatives, some children continue…

Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity
 Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Researchers in the NEPTUNE network and collaborators at Nationwide Children’s Hospital have confirmed their preclinical studies demonstrating a direct correlation between the severity of disease and prothrombotic biomarkers in patients with nephrotic syndrome. Nephrotic syndrome (NS) dramatically increases the risk of a life-threatening blood clot, but until recently, clinicians have had no reasonable biomarker to…

Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors
 Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors 772 447 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Survival has dramatically improved for numerous pediatric cancers over the last several decades, with a notable and very deadly exception: diffuse intrinsic pontine glioma (DIPG). Now, a community of researchers and clinician-scientists have set the stage for a renewed — and better-armed — assault against this beast of a brain tumor. The past 50 years…

Understanding Immune Responses to Build a Better Vaccine
 Understanding Immune Responses to Build a Better Vaccine 1024 606 Abbie Miller
By: Abbie Miller

Researchers have used studies of respiratory syncytial virus and infant immune responses to develop a promising vaccine candidate. Respiratory syncytial virus (RSV) is an incredibly common yet potentially deadly pathogen. Almost everyone becomes infected with RSV during their first three years of life, but for certain populations — infants and elderly or immunocompromised people —…

Even Severe Asthma Can Improve if Guidelines Are Followed
 Even Severe Asthma Can Improve if Guidelines Are Followed 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Patients treated with adequate medications and asthma family education can significantly improve irrespective of asthma severity. Severe asthma accounts for 5-8% of patients with asthma, but this group is more challenging to treat and is responsible for up to 40% of total asthma-care expenses. The majority of patients with severe asthma have difficult-to-treat asthma (in…

Global Study Highlights Antibiotic Overuse in the NICU
 Global Study Highlights Antibiotic Overuse in the NICU 1024 683 Abbie Miller
By: Abbie Miller
Baby in NICU

Antimicrobial stewardship programs were associated with lower antibiotic use, regardless of the country’s income level. Excessive antibiotic use among infants born preterm in the newborn intensive care unit (NICU) is associated with poor patient outcomes, such as sepsis, necrotizing enterocolitis, bronchopulmonary dysplasia and others, and contributes to the emergence of multi-drug resistant microbes. A new…

Can Neurocognitive Functioning Tests Help Predict Future Suicide Attempts?
 Can Neurocognitive Functioning Tests Help Predict Future Suicide Attempts? 1024 683 Mary Bates, PhD
By: Mary Bates, PhD
Sad black teenage girl

Researchers identify sex-specific deficits in measures of working memory and affective processing associated with suicidal behavior in youth with depression. To prevent youth suicide, researchers are working to identify factors associated with suicide attempts in adolescents at elevated risk for suicidal behavior. In a new study, researchers from Nationwide Children’s examined neurocognitive functioning in suicidal…

Lower Traffic Volumes During COVID-19 Shut-Downs Lead to Fewer Injuries
 Lower Traffic Volumes During COVID-19 Shut-Downs Lead to Fewer Injuries 1024 683 Laura Dattner
By: Laura Dattner
Teen driving a car

To minimize transmission of COVID-19, in spring 2020, most U.S. states passed policies promoting social distancing through stay-at-home orders prohibiting non-essential travel. While vehicle-miles traveled in the United States decreased by 41% in April 2020 compared to 2019, the effect of this mobility decrease on motor vehicle crashes (MVCs) is poorly understood. Motao (Matt) Zhu,…

Innovative Biostatistical Methodology for Causal Evaluation of Medical Treatments
 Innovative Biostatistical Methodology for Causal Evaluation of Medical Treatments 150 150 Laura Dattner
By: Laura Dattner

Medical records are readily available nowadays, but using medical records to evaluate medical treatments is full of challenges. Henry Xiang, MD, MPH, PhD, professor of medicine and principal investigator in the Center for Injury Research and Policy, collaborated with biostatisticians from The Ohio State University College of Public Health to develop a novel statistical method…

Introducing a New SARS-CoV-2 Vaccine Candidate
 Introducing a New SARS-CoV-2 Vaccine Candidate 1024 512 Abbie Miller
By: Abbie Miller
coronavirus

The new vaccine candidate takes advantage of the long and successful history of the measles vaccine. A team of researchers from The Ohio State University and Nationwide Children’s have built a novel vaccine candidate against SARS-CoV-2. The candidate, published in Proceedings of the National Academy of Sciences (PNAS), used the measles vaccine as a vector…

Calls to Poison Centers About High-Powered Magnets Increased by 444% After Ban Lifts
 Calls to Poison Centers About High-Powered Magnets Increased by 444% After Ban Lifts 1024 521 Laura Dattner
By: Laura Dattner
cube made of ball magnets in the palm of a hand

High-powered magnets are small, shiny and made from powerful rare earth metals. Since they started showing up in children’s toys in the early 2000s and then later in desk sets in 2009, high-powered magnets have caused thousands of injuries and are considered to be among the most dangerous ingestion hazards in children. When more than one…

Girls’ Perineal Microbiomes Change Over Development, After Urinary Tract Infection
 Girls’ Perineal Microbiomes Change Over Development, After Urinary Tract Infection 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Next-generation sequencing reveals different flora associated with developmental milestones in girls, as well as disruptions in patients with a history of urinary tract infection. In a new pilot study, researchers from Nationwide Children’s Hospital evaluated the microbiomes of girls at specific developmental timepoints. They found shifts in the perineal microbiome corresponding with important developmental milestones,…

Immersive Virtual Reality-Based Training Improves Empathy Among Health Care Providers
 Immersive Virtual Reality-Based Training Improves Empathy Among Health Care Providers 414 224 Lauren Dembeck
By: Lauren Dembeck

A novel VR-based training program helps providers develop knowledge, skills, and attitudes to understand how social determinants of health affect patients and how to mitigate their effects. Researchers recently reported their novel virtual reality (VR)-based empathy training program, Making Professionals Able THrough Immersion (MPATHI), improved knowledge, skills, and attitudes of health care providers for care…

No More Sitting in the Dark?
 No More Sitting in the Dark? 1024 728 Laura Dattner
By: Laura Dattner

A new study from the Center for Injury Research and Policy, Sports Medicine and Emergency Medicine is the first to document objectively self-paced physical and cognitive activity post-concussion among youth. The results suggest they may be able to engage in physical and cognitive activity as soon as tolerated post-concussion. Clinical guidelines for managing concussion in…

Modifying NK Cells With CRISPR/Cas9
 Modifying NK Cells With CRISPR/Cas9 969 533 Mary Bates, PhD
By: Mary Bates, PhD
Illustration of NK Cells, T Cells, other immune cells floating across white background

A proof-of-concept study shows success in modifying NK cells for multiple myeloma. In a new proof-of-concept study, researchers from Nationwide Children’s Hospital used CRISPR/Cas9 technology to genetically modify natural killer immune cells, which they then showed are able to address a recognized hurdle in immunotherapy of multiple myeloma. “It was an amazing feeling when we…

Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer
 Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Evidence from a new study highlights the importance of family-centered decision processes and clinician facilitation in improving fertility preservation uptake prior to cancer treatment. Numerous cancer treatments can reduce fertility or render some patients entirely infertile, but fertility preservation (FP) services have historically had low rates of uptake. When a young person is diagnosed with…

A Quick and Reliable Method to Determine The Time to Full Weight Bearing in Patients Undergoing Intramedullary Limb Lengthening
 A Quick and Reliable Method to Determine The Time to Full Weight Bearing in Patients Undergoing Intramedullary Limb Lengthening 1024 575 Lauren Dembeck
By: Lauren Dembeck

A new method to objectively measure bone healing may help the patient with an intramedullary lengthening nail make strides to full weight bearing status. Intramedullary limb lengthening is indicated for the treatment of limb length discrepancy and short stature. Traditionally, the procedure was done using external fixators, which must remain on the patient’s limb for…

No Evidence Supporting Prophylactic Indomethacin Treatment for Most Preterm Infants
 No Evidence Supporting Prophylactic Indomethacin Treatment for Most Preterm Infants 1024 575 Lauren Dembeck
By: Lauren Dembeck
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

Inconsistency among studies prompted researchers to take a closer look at the drug’s effects. Despite limited evidence supporting its use, prophylactic indomethacin treatment is often administered to very preterm infants within the first 24-hours after birth to reduce the risks of intraventricular hemorrhage and longer-term neonatal morbidities, such as bronchopulmonary dysplasia (BPD). Data from a…

Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling
 Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Researchers have uncovered a rare subset of meningiomas with a genetic driver shared by another cancer type, opening the door to new therapeutic considerations. When an interesting or intractable cancer case arises at Nationwide Children’s Hospital, researchers and clinicians involved in the Brain Tumor Protocol through The Steve and Cindy Rasmussen Institute for Genomic Medicine…

Batten Disease Experts Publish Common Key Practices For Enzyme Replacement Therapy
 Batten Disease Experts Publish Common Key Practices For Enzyme Replacement Therapy 1024 683 Lauren Dembeck
By: Lauren Dembeck

Neuronal ceroid lipofuscinosis type 2 disease is a rare, rapidly-progressing, neurodegenerative lysosomal storage disorder caused by tripeptidyl peptidase 1 deficiency. This form of Batten disease has an onset between two to four years of age. Children with CLN2 disease may experience intractable epilepsy, progressive cognitive and motor decline, and loss of vision. Historically, most children…

Polysomnography in Infants: How Much Total Sleep Time is Really Needed?
 Polysomnography in Infants: How Much Total Sleep Time is Really Needed? 1024 575 Abbie Miller
By: Abbie Miller
small baby with nose canula

Study identifies factors affecting total sleep time and suggests shorter sleep times can be effective for accurate interpretation and clinical decision making. In a recent study published in American Journal of Perinatology, experts from Nationwide Children’s Hospital investigated factors that affect total sleep time(TST) during infant polysomnography (PSG) in an effort to determine how much TST is…

Risk Stratification in Infants With Primary Pulmonary Vein Stenosis
 Risk Stratification in Infants With Primary Pulmonary Vein Stenosis 1024 681 Mary Bates, PhD
By: Mary Bates, PhD
sleeping infant

Categorizing infants with pulmonary vein stenosis into stable and progressive groups could help inform treatment. Primary pulmonary vein stenosis is a rare, often lethal, cardiac disease. It is challenging to treat, as the disease can be progressive in some patients but not others. In a new study, researchers from Nationwide Children’s describe outcomes among preterm infants…

Cerebral Organoids Provide Insight into Human Brain Development and Neurological Disease
 Cerebral Organoids Provide Insight into Human Brain Development and Neurological Disease 1024 575 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM
microscopic image of brain organoid

Correlations between the neuroelectrical maturation and cellular development of cerebral organoids highlight the organoids’ translational and therapeutical potential in early neurodevelopmental disorders. Cerebral organoids are three-dimensional miniature organs that resemble the human brain. Derived from human pluripotent stem cells, cerebral organoids have emerged to advance stem cell research, improve three-dimensional tissue culture techniques and enhance…

Prematurity and Genomics: Can Complications For a Baby Born Preterm Be Predicted?
 Prematurity and Genomics: Can Complications For a Baby Born Preterm Be Predicted? 1024 575 Eric Butterman
By: Eric Butterman
conceptual art of DNA

Complications from being born preterm are the number one cause of death in the world for children under the age of five, says Leif Nelin, MD, division chief of Neonatology at Nationwide Children’s Hospital and professor of Pediatrics at The Ohio State University . But why do some babies born preterm develop complications while many others seem…

Advances in Neonatal GERD
 Advances in Neonatal GERD 1024 683 Mary Bates, PhD
By: Mary Bates, PhD
Dr. Jadcherla

New studies from the Jadcherla Lab provide insights into diagnosing, classifying and treating GERD in infants. Differentiating gastroesophageal reflux (GER), which is defined as the passage of gastric contents into the esophagus, from GER disease (GERD), when reflux is associated with troubling symptoms, remains a challenge in infants. Symptom-based diagnosis and treatment of GERD has…

Krabbe Disease Successfully Treated With Gene Therapy in Preclinical Animal Model
 Krabbe Disease Successfully Treated With Gene Therapy in Preclinical Animal Model 150 150 Lauren Dembeck
By: Lauren Dembeck

Gene therapy shows promise for clinical benefit in demyelinating, neurodegenerative disease. Krabbe disease is an aggressive, incurable pediatric neurodegenerative disease caused by mutations in the galactosylceramidase (GALC) gene. Deficiency of the GALC protein activity leads to cytotoxic accumulation of a cellular metabolite called psychosine, which compromises normal turnover of myelin in the central and peripheral…

New Newborn Screening Approach Allows for Earlier Detection of Menkes Disease
 New Newborn Screening Approach Allows for Earlier Detection of Menkes Disease 1024 575 Natalie Wilson
By: Natalie Wilson

Targeted next generation DNA sequencing can enable improved clinical outcomes for any genetic disorder not detectable through other biochemical newborn screening methods. Menkes disease is a rare, X-linked recessive pediatric disease caused by gene mutations of the copper transporter gene, ATP7A, leading to low levels of copper in infants’ blood and brain, as well as abnormal…

An Expanded, Multicenter Look at Gene Therapy for Spinal Muscular Atrophy
 An Expanded, Multicenter Look at Gene Therapy for Spinal Muscular Atrophy 1024 575 Mary Bates, PhD
By: Mary Bates, PhD
Colorful illustration of gene therapy in action

New study confirms safety and efficacy in children under two years old. In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been…

How Do Parents and Surgeons Evaluate Treatment Options for Uncomplicated Appendicitis?
 How Do Parents and Surgeons Evaluate Treatment Options for Uncomplicated Appendicitis? 1024 575 Natalie Wilson
By: Natalie Wilson
Parent and provider at child's hospital bed

The most recent in a series of publications on management of uncomplicated appendicitis showed patients and families cited the same concerns when electing either surgery or nonoperative treatment. Antibiotics alone have been shown to successfully treat uncomplicated appendicitis in children. But patients, families and surgeons may differently evaluate risks and benefits of this treatment option.…

Novel Intervention Helps Infants With Cerebral Palsy Develop Arm and Hand Function
 Novel Intervention Helps Infants With Cerebral Palsy Develop Arm and Hand Function 1024 683 Abbie Miller
By: Abbie Miller

A new NIH-funded randomized controlled trial shows that an intervention combining a patented soft restraint harness, therapist coaching and parent training increases reach smoothness, fine motor skills and tactile sensation in the more-affected upper extremity. For the first time in infants with CP under 2, a clinical trial of this type of intervention was shown…

Race Is a Risk Factor for Postoperative Death in Apparently Healthy Children in United States
 Race Is a Risk Factor for Postoperative Death in Apparently Healthy Children in United States 1024 683 Abbie Miller
By: Abbie Miller

African American children were nearly 3.5 times more likely to die within 30 days after surgery, compared to white peers. In a new study, published in Pediatrics, researchers have shown that being African American was strongly associated with a higher risk of postoperative complications and mortality among apparently healthy children. In fact, compared to their white peers,…

Targeting Bacterial Biofilm Linchpin Prevents and Treats Recalcitrant Biofilm-Mediated Infections
 Targeting Bacterial Biofilm Linchpin Prevents and Treats Recalcitrant Biofilm-Mediated Infections 1024 575 Abbie Miller
By: Abbie Miller
Illustration showing the precise pattern of a biofilm - a perfect, 3D matrix

A new study highlights two approaches with substantive efficacy and potential for broad application to combat biofilm-mediated diseases. Chronic and recurrent bacterial diseases are treatment-resistant due to the ability of the pathogens to establish biofilms, which act as fortresses built of extracellular DNA and proteins to protect populations of the bacteria. For more than 11…

Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient
 Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient 1024 575 Lauren Dembeck
By: Lauren Dembeck

Vedolizumab is a monoclonal antibody that targets α4β7 integrin present on gut homing T cells and is indicated for adult inflammatory bowel disease Physicians at Nationwide Children’s Hospital have successfully treated steroid-refractory gastrointestinal acute graft-versus-host disease (SR GI aGVHD) with adjuvant vedolizumab therapy in a pediatric allogeneic hematopoietic cell transplant (HCT) recipient. The case was…

Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD
 Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD 150 150 Natalie Wilson
By: Natalie Wilson

Researchers explore gene responses among SCD patients hospitalized for acute complications to inform understandings of the under-studied disease. Sickle cell disease (SCD) is a chronic, genetic disorder characterized by structural changes in circulating red blood cells. According to the CDC, SCD affects approximately 100,000 individuals in the United States alone and accounts for 3,000 births…

Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children With Duchenne Muscular Dystrophy
 Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children With Duchenne Muscular Dystrophy 150 150 Abbie Miller
By: Abbie Miller

One-year data from the first four patients to receive a single dose of the rAAVrh74.MHCK7.micro-dystrophin gene therapy is published in JAMA Neurology. Researchers from Nationwide Children’s Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy…

Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy
 Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy 720 480 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Research looking at mitochondrial targets in kidney health holds promise for cisplatin-induced kidney injury. Recent work exploring the mitochrondrial metabolism in the kidneys following exposure to cisplatin, a common chemotherapy, has revealed a key role of superoxide (O2•-, an indicator of oxidative stress in renal cells associated with cell damage and death) in both acute…

Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden
 Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden 1024 495 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
blood cells

Clinical trials using gene therapy to boost the body’s production of clotting factor aim to remove the need for regular infusions — ideally giving patients years or even decades free from daily worry about their condition. Unlike most pediatric treatment centers, Nationwide Children’s Hospital is one of only a few dozen sites worldwide selected to…