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Abbie Miller

1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients
 1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients 1024 614 Abbie Miller
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abstract art of magnifying glass over DNA strand

Data from the analysis helps clinicians confirm diagnoses and identify targeted treatments. The data also support new pediatric cancer research through the National Cancer Institute’s Childhood Cancer Data Initiative.   The CCDI Molecular Characterization Initiative (MCI) is a project that aims to collect, analyze and report clinical and molecular data and is currently supporting Children’s…

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Tissue Engineering and Fetal Medicine: A New Frontier for Congenital Heart Disease
 Tissue Engineering and Fetal Medicine: A New Frontier for Congenital Heart Disease 1024 575 Abbie Miller
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Fetus in utero receiving valvuloplasty

Procedures that utilize cardiac catheterization to improve fetal heart development are often successful, but they are not without risk. And even if they can successfully prevent the development of single ventricle disease, there is always ongoing heart valve disease (HVD). HVD may not be as life-threatening as single ventricle disease, but it requires lifelong care.…

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What Pediatricians Need to Know About Lymphedema in Pediatrics
 What Pediatricians Need to Know About Lymphedema in Pediatrics 150 150 Abbie Miller
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Lymphedema is chronic, progressive swelling caused by maldevelopment or disruption of the lymphatic system. Typically, patients present with an asymmetric swelling of an arm or leg that progressively worsens. Min-Jeong Cho, MD, a microsurgeon and assistant professor at The Ohio State University and Nationwide Children’s Hospital, says that lymphedema affects more children and teens than…

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Micro-dystrophin: A Small Gene With Big Promise
 Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller
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SRP-9001 for Duchenne muscular dystrophy supplies a functional dystrophin gene via AAVrh74 gene therapy. In 1969, Jerry Mendell, MD, was working at the National Institute of Neurological Disorders and Stroke (NINDS) when he saw his first patient with Duchenne muscular dystrophy (DMD). DMD, a severe form of muscular dystrophy caused by a mutation in the…

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Using Machine Learning in the Electronic Medical Record to Save Lives
 Using Machine Learning in the Electronic Medical Record to Save Lives 1024 683 Abbie Miller
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physicians in white coats looking at a tablet

The deterioration risk index identifies patients at risk for deterioration and poor outcomes, triggering the care team to act before a crisis happens. In a report published in Pediatric Critical Care Medicine, a team from Nationwide Children’s Hospital describes a machine learning tool for timely identification of hospitalized children at risk for deterioration – a…

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Pediatric Obesity Can Be Treated Safely and Effectively
 Pediatric Obesity Can Be Treated Safely and Effectively 1024 575 Abbie Miller
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New guidelines from the American Academy of Pediatrics emphasize evidence for safe, effective treatment of pediatric obesity for long-term benefit of children and adolescents.   More than 14.4 million U.S. children and adolescents have obesity, making it one of the most common chronic conditions facing American youth and families. Obesity is a chronic disease associated…

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5 Things to Know About Prune Belly Syndrome
 5 Things to Know About Prune Belly Syndrome 1024 683 Abbie Miller
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Linda Baker, MD

Linda Baker, MD, urologist, principal investigator, and one of the world’s foremost experts on prune belly syndrome, recently joined Nationwide Children’s Hospital as the co-director of the Kidney and Urinary Tract Center. She shares some important things to know about this rare disease. 1. Prune belly is a rare, congenital malformation. Prune belly syndrome (PBS)…

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New Guidelines for Care of Children With Cerebral Palsy
 New Guidelines for Care of Children With Cerebral Palsy 1024 683 Abbie Miller
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Young child with Cerebral Palsy

Guidelines from the American Academy of Pediatrics and the American Academy for Cerebral Palsy and Developmental Medicine highlight advances in diagnosis, care and outcomes for children and adolescents with cerebral palsy. In late 2022, the American Academy of Pediatrics and the American Academy for Cerebral Palsy and Developmental Medicine released new guidelines to help primary…

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Predicting Risk of Nosocomial Infection in Pediatric Burn Patients: Can Measures of Adaptive Immune Function Help?
 Predicting Risk of Nosocomial Infection in Pediatric Burn Patients: Can Measures of Adaptive Immune Function Help? 150 150 Abbie Miller
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Pediatric burn injuries affect 1 million children each year in the United States. While burn care improvements have decreased mortality, infection-related complications remain a significant problem. According to the National Burn Repository, four of the top five complications of pediatric burns are infection related. Plus, infection-related complications occur in up to 60% of patients with…

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New Guidelines Offer a Clear Path for Expandable Endoprosthesis Use in Post-Sarcoma Limb Reconstruction
 New Guidelines Offer a Clear Path for Expandable Endoprosthesis Use in Post-Sarcoma Limb Reconstruction 150 150 Abbie Miller
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Limb salvage operation is the standard of care for pediatric extremity bone sarcomas. These tumors often occur during the second decade of life, when children are growing rapidly. Reconstructive surgeries must account for anticipated remaining growth and the potential for resulting limb inequity. Osteosarcoma and Ewing sarcoma are the most common primary malignant bone tumors,…

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Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure
 Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

By binding to DNA in the nucleus, the Ewing sarcoma-specific transcription factor EWS/FLI changes chromatin organization, leading to altered gene expression and the development of Ewing sarcoma.   A new report, published in Nucleic Acids Research, focuses on how the Ewing sarcoma-specific transcription factor EWS/FLI changes spatial organization of DNA in the nucleus of Ewing…

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New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene
 New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene 1024 575 Abbie Miller
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A novel missense mutation in identical twins leads to discovery and definition of new inherited disorder of brain copper metabolism. Copper is essential for many cellular functions, including cellular respiration, antioxidant defense, neurotransmitter biosynthesis and neuropeptide amidation, among others. Until recently, only two inborn errors of copper metabolism were well established. Both are rare. Wilson’s…

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Cardiopulmonary Rehabilitation Post-COVID: Could Your Patient Benefit?
 Cardiopulmonary Rehabilitation Post-COVID: Could Your Patient Benefit? 1024 728 Abbie Miller
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The road to recovery from COVID-19, the disease caused by SARS-CoV-2, is not always smooth, short or straight. For adults and children, long COVID, a condition where symptoms such as fatigue, headache and decreased respiratory function persist after the virus has “cleared” the body, can add increased stress and hardship. Additionally, even mild cases of…

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BRUE: What Is It? How Is It Treated?
 BRUE: What Is It? How Is It Treated? 1024 575 Abbie Miller
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BRUE –brief resolved unexplained event – whether you pronounce it brew-EE or brOO, it might be a term you haven’t run across since medical school. In a recent resident-led episode of PediaCast CME, Meghan Fennell, MD, and Vanessa Thiel, MD, break down everything you need to know about BRUE. Listen to the PediaCast CME Episode…

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The True History of the Nuremberg Code: Leo Alexander’s Blueprint Uncovered
 The True History of the Nuremberg Code: Leo Alexander’s Blueprint Uncovered 150 150 Abbie Miller
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An investigation of Duke University’s Medical Center’s Archives boxes labeled “Alexander’s Papers” reveals a document containing six principles, which were included nearly word-for-word in the Nuremberg Code.  At the end of the famous Doctors’ Trial, the trial of Nazi doctors for their crimes and treatment of people who were identified as “research participants,” Judge Sebring…

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When Functional Outcomes Are Achieved Post-Op, Do Patients and Families Care About Scarring?
 When Functional Outcomes Are Achieved Post-Op, Do Patients and Families Care About Scarring? 1024 683 Abbie Miller
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A recent editorial from Dr. Daniel DaJusta questions whether cosmetic concerns after surgery are primarily driven by surgeons, patients or parents. It is widely accepted that having a smaller, neater or less visible scar following surgery is a preferable cosmetic outcome to a large, ragged or highly visible scar. This is supported anecdotally by surgeons,…

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Moving Emergency Medicine Research Forward
 Moving Emergency Medicine Research Forward 1024 614 Abbie Miller
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Utilizing exception from informed consent studies and a national network of emergency medicine experts, clinician-scientists are advancing research to uncover best practices and improve care. Exception from informed consent (EFIC) is a special rule set utilized by the U.S. Food and Drug Administration (FDA) to support research in emergency situations. The word exception is particularly…

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First In Human
 First In Human 1024 752 Abbie Miller
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Top-down view of heart valves

The Heart Center at Nationwide Children’s Hospital has a long history of helping to bring innovations from the lab to the patient. The latest, the Autus Valve, aims to improve care and outcomes for children with pulmonary valve disease. In December 2021, Mark Galantowicz, MD, cardiothoracic surgeon and director of The Heart Center at Nationwide…

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Novel Digital Storybook Intervention Aims to Support Children With Hearing Loss
 Novel Digital Storybook Intervention Aims to Support Children With Hearing Loss 1024 683 Abbie Miller
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Hear Me Read, invented and developed at Nationwide Children’s Hospital, is now being validated in a prospective clinical trial. For children who are deaf or hard of hearing, access to sound via hearing aids and cochlear implants and intensive speech therapy with highly trained verbal therapists are important parts of developing speech and literacy. “Children…

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Improving Neuro-Critical Care Outcomes for Children in Sub-Saharan Africa
 Improving Neuro-Critical Care Outcomes for Children in Sub-Saharan Africa 1024 683 Abbie Miller
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Nicole O'Brien, MD

Nicole O’Brien, MD, is working with health experts in sub-Saharan Africa to develop Centers of Excellence for the use of transcranial doppler ultrasound (TCD).   The first time Nicole O’Brien went to Africa, she was not a doctor. In her words, she “wasn’t even close to medical.” She was staying in a small rural village…

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NCHart-1 a New Approach to Estimating Total Body Surface Area Burn Percentages
 NCHart-1 a New Approach to Estimating Total Body Surface Area Burn Percentages 150 150 Abbie Miller
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The new chart reduces math errors, improving accuracy for better outcomes. When a child has a burn injury, immediate care is essential. Fluid administration and treatment resources needed are decisions made in the field by emergency medical technicians (EMTs) and paramedics. Both decisions hinge on what percent of the child’s skin has burns. This number…

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New Approach to Dystrophin Quantification Can Help Researchers Studying Muscular Dystrophy
 New Approach to Dystrophin Quantification Can Help Researchers Studying Muscular Dystrophy 150 150 Abbie Miller
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Dystrophin is the protein product of the DMD gene and plays an important role in Becker and Duchenne muscular dystrophies. Researchers in the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital have developed an automated, unbiased approach for the precise quantification of dystrophin immunofluorescence in muscle samples. Dystrophin is…

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Creating a Physician Workforce that Reflects the Patients and Families We Serve
 Creating a Physician Workforce that Reflects the Patients and Families We Serve 1024 683 Abbie Miller
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Workforce disparities persist within health care institutions and medical training. While individuals who identify as Black/African American, Latinx/Hispanic, Native American, and Pacific Islander comprise roughly 30% of the U.S. population, they are less than 15% of physicians, making them underrepresented in medicine – or URM. In fact, as the U.S. population grows more diverse, the…

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Gene Therapy Rescues Phenotype in Preclinical Model for Limb Girdle Muscular Dystrophy 2A/R1
 Gene Therapy Rescues Phenotype in Preclinical Model for Limb Girdle Muscular Dystrophy 2A/R1 1024 575 Abbie Miller
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Replacement of CAPN3 gene via AAV-mediated gene therapy resulted in significant, robust improvements in functional outcomes and muscle physiology in the preclinical model. LGMDs are a rare form of muscular dystrophy for which there is no treatment. As a heterogenous group caused by a number of mutations, the complexity of classification makes the disease challenging…

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Repeated Injury to Stem Cells Could be a Significant Contributor to Chronic Lung Disease
 Repeated Injury to Stem Cells Could be a Significant Contributor to Chronic Lung Disease 1024 575 Abbie Miller
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Illustration of lungs on blue silhouette of upper chest on black background

A series of experiments shows that biological aging of tracheobronchial stem cells could drive the development of chronic lung disease. In a recent publication in STEM CELLS Translational Medicine (SCTM), researchers showed that repeated injury to epithelial cells in the airway caused biological aging of the stem cells located there. Tracheobronchial stem cells (TSCs) are…

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Teens on the Road: How Technology, Policy and Parents Influence Driving Safety
 Teens on the Road: How Technology, Policy and Parents Influence Driving Safety 1024 633 Abbie Miller
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Traffic crashes are a leading cause of death in the United States. For teens, the stakes are especially high.

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Unraveling the Role of Pioneer Factors in Childhood Cancer
 Unraveling the Role of Pioneer Factors in Childhood Cancer 150 150 Abbie Miller
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New study implicates PAX3-FOXO1, an oncogenic driver of rhabdomyosarcoma, as a chimeric pioneer factor. Transcription factors are proteins that read the DNA sequences and can direct the transcription of that sequence into mRNA for translation into a protein. Transcription can become deregulated in cancer. Normally, transcription factors read sequences of DNA that are described as…

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Robotic Surgery Is Here to Stay
 Robotic Surgery Is Here to Stay 150 150 Abbie Miller
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A recent publication describes increases in the use of robotic surgery in children’s hospitals across the country.  The use of robotic surgery in tertiary children’s hospitals has been on the rise since the 2000s. In a recent publication, researchers from Nationwide Children’s Hospital sought to characterize the use and growth in acceptance of robotic surgeries…

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Providing Education and PrEP for Teens at Risk for HIV
 Providing Education and PrEP for Teens at Risk for HIV 1024 683 Abbie Miller
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In a recent PediaCast CME, Mike Patrick, MD, and Megan Brundrett, MD, share important information about offering PrEP and HIV-related education in your primary care practice. Listen to the Full Episode About 20% of new cases of HIV, the virus that causes Acquired Immunodeficiency Syndrome (AIDS), are occurring in youth aged 13 to 24. HIV/AIDS…

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

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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
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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…

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9 Facts About the COVID-19 Vaccine
 9 Facts About the COVID-19 Vaccine 1024 683 Abbie Miller
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COVID-19 is a disease caused by the SARS-CoV-2 virus. In the United States, three vaccines are approved for emergency use, those produced by Pfizer, Moderna and Johnson & Johnson (J&J). These vaccines dramatically reduce your risk of getting severely ill or dying from COVID-19. The vaccines are also being shown to reduce the spread of…

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How Can We Make Child Health Equitable?
 How Can We Make Child Health Equitable? 550 350 Jeb Phillips, Abbie Miller and Natalie Wilson
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Researchers in the Center for Child Health Equity and Outcomes Research at Nationwide Children’s Hospital are tackling this question by identifying health disparities and uncovering exactly how social drivers of health impact outcomes. “Health equity means that everyone has a fair and just opportunity to be as healthy as possible.” That’s how the Robert Wood…

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Understanding Immune Responses to Build a Better Vaccine
 Understanding Immune Responses to Build a Better Vaccine 1024 606 Abbie Miller
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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 —…

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Global Study Highlights Antibiotic Overuse in the NICU
 Global Study Highlights Antibiotic Overuse in the NICU 1024 683 Abbie Miller
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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…

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Introducing a New SARS-CoV-2 Vaccine Candidate
 Introducing a New SARS-CoV-2 Vaccine Candidate 1024 512 Abbie Miller
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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…

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Targeting FSHD With Designed Antisense RNAs
 Targeting FSHD With Designed Antisense RNAs 1024 683 Abbie Miller
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Researchers describe small nuclear RNA antisense expression cassettes as a way to silence the DUX4 gene underlying facioscapulohumeral muscular dystrophy. Facioscapulohumeral muscular dystrophy (FSHD) arises from genetic and epigenetic changes that result in expression of the DUX4 gene in muscle. DUX4 is a developmental gene that is normally off in healthy muscle.  When expressed in…

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Incidence and Impact of Acute Kidney Injury in Patients With Hypoplastic Left Heart Syndrome
 Incidence and Impact of Acute Kidney Injury in Patients With Hypoplastic Left Heart Syndrome 150 150 Abbie Miller
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While incidence of severe kidney injury after surgery for hypoplastic left heart syndrome is low, it may be linked to increased 30-day mortality. Acute kidney injury – the abrupt decline of renal function – following pediatric heart surgery leads to worse outcomes, including increased mortality. The overall incidence of acute kidney injury (AKI) after pediatric…

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Lauren Bakaletz Named 2021 Allen Distinguished Scholar in Pediatric Research
 Lauren Bakaletz Named 2021 Allen Distinguished Scholar in Pediatric Research 1024 575 Abbie Miller
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Lauren Bakaletz, PhD, director of the Center for Microbial Pathogenesis in the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital, was named the 2021 Allen Distinguished Scholar in Pediatric Research. The award is given in honor of former Nationwide Children’s CEO, Steve Allen, MD, and his role in growing the AWRI into a preeminent research institution.…

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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
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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…

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What Are the Clinical Implications of Crescentic Glomerulonephritis?
 What Are the Clinical Implications of Crescentic Glomerulonephritis? 1024 732 Abbie Miller
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The Pediatric Nephrology Research Consortium’s most recent study investigates the risk factors for disease progression in children with glomerulonephritis and crescents on kidney biopsy. In patients with glomerulonephritis (GN), the histopathology of the kidney may include crescents, which result from extra-capillary proliferation of cells within the glomerulus. A recent study from the Pediatric Nephrology Research…

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Measuring the Progression of CF in School-Aged Children
 Measuring the Progression of CF in School-Aged Children 1024 575 Abbie Miller
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Illustration of lungs on blue silhouette of upper chest on black background

A multicenter study utilized chest computed tomography and lung function tests taken repeatedly over two years in school-aged children with mild cystic fibrosis. Understanding disease progression in chronic illnesses is important to managing patients and improving outcomes. For children with cystic fibrosis, it can be difficult to predict how long it will take for negative…

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More to Telehealth Than Meets the Screen
 More to Telehealth Than Meets the Screen 1024 535 Abbie Miller
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Collage of health technology tools

Telehealth has become essential to American health care during the COVID-19 pandemic, but is it really the solution to our biggest access-to-care problems? When COVID-19 ignited stay-at-home orders, public and private insurers quickly relaxed the rules for covering telehealth visits. Health care systems responded in kind by rapidly expanding their telehealth capacity and training. Expanding…

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Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration
 Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration 1024 683 Abbie Miller
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Alteration in the Lymphatic System Leading to Flow Disorders The lymphatic system plays a critical role in circulating a clear to yellow-colored fluid called lymph, which contains proteins, immune factors and cells throughout the body. It is also responsible for collecting fats and fat-soluble vitamins from the gastrointestinal tract in cloudy-appearing chylous fluid. This fluid…

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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
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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…

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Additions to Neuro-Oncology Team Bring CONNECT Consortium to Nationwide Children’s
 Additions to Neuro-Oncology Team Bring CONNECT Consortium to Nationwide Children’s 1024 575 Abbie Miller
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Maryam Fouladi, MD, has been named the co-executive director of the Pediatric Neuro-Oncology Program at Nationwide Children’s Hospital and Rachid Drissi, PhD, will join the Center for Childhood Cancer and Blood Diseases in the Abigail Wexner Research Institute as a principal investigator focused on cellular aging and telomerase inhibitors to suppress brain tumor growth in children. Dr. Fouladi will continue her…

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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
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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,…

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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
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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…

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From Expectant Parents to Advocates: One Family’s Journey With Hypoplastic Left Heart Syndrome
 From Expectant Parents to Advocates: One Family’s Journey With Hypoplastic Left Heart Syndrome 1024 575 Abbie Miller
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Mila’s parents wanted her to have the best chance at surviving her high-risk comprehensive stage 2 surgery – their journey led them to The Heart Center at Nationwide Children’s. Mid-way into their pregnancy, Jacob and Kayla went in for their 20-week ultrasound. “We were very excited to get into things like paint colors for our…

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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
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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…

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