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Lauren Dembeck

Beyond Modulators: Ensuring All Patients With Cystic Fibrosis Benefit From the Next Wave of Therapy
 Beyond Modulators: Ensuring All Patients With Cystic Fibrosis Benefit From the Next Wave of Therapy 1024 618 Lauren Dembeck
By: Lauren Dembeck
Two smiling young children sit side by side indoors, facing the camera, with a colorful blurred artwork in the background.

Before CFTR modulators transformed care, cystic fibrosis (CF) was defined by relentless daily treatment and progressive lung disease. Children grew up with thick airway secretions, chronic cough, recurrent pulmonary infections, and frequent hospitalizations. Maintaining weight was a constant struggle, and lung function typically declined year after year despite aggressive airway clearance, antibiotics and nutritional support.…

How Diabetes Rewires the Heart’s Smallest Vessels
 How Diabetes Rewires the Heart’s Smallest Vessels 1024 597 Lauren Dembeck
By: Lauren Dembeck
Microscopy image of a mouse heart sample colored with red and green fluorescent dye, and marked for single-cell sampling.

A landmark study uncovers how diabetes alters the heart at the cellular level. Cardiovascular disease is the leading cause of death in type 2 diabetes. While its effects on large arteries are well recognized, growing evidence suggests the earliest cardiovascular injury occurs in the heart’s smallest vessels. Coronary microvascular disease (CMD), marked by impaired endothelial…

New Health Challenges in the Post-CFTR Modulator Era
 New Health Challenges in the Post-CFTR Modulator Era 1024 576 Lauren Dembeck
By: Lauren Dembeck

The remarkable progress in CF care has also revealed new challenges. As individuals with CF live longer, clinicians are seeing higher frequencies of complications and other down- stream health concerns that rarely emerged in the past and are now focused on aging-related diseases and maintaining their overall health. Karen McCoy, MD, pediatric pulmonologist and renowned…

The Growing Use of Robotics in General Pediatric Surgical Procedures
 The Growing Use of Robotics in General Pediatric Surgical Procedures 1024 683 Lauren Dembeck
By: Lauren Dembeck

Pediatric surgeons are increasingly turning to robotic technology for the delivery of safe and enhanced minimally invasive surgical approaches for younger children. Over the past two decades, the use of minimally invasive surgery (MIS) techniques for both laparoscopic and thoracoscopic surgery have been widely adopted for the treatment of children requiring various surgical procedures. This…

DNA Methylation-Based Diagnostics: Refining Diagnosis for the Most Complex Pediatric Brain Tumors
 DNA Methylation-Based Diagnostics: Refining Diagnosis for the Most Complex Pediatric Brain Tumors 1024 783 Lauren Dembeck
By: Lauren Dembeck

Developed at Nationwide Children’s, the MACDADI classifier is compatible with next-generation methylation arrays and quickly delivers more accurate tumor diagnoses, offering a clinically validated alternative to outdated and unregulated methylation classifiers. Diagnosing central nervous system (CNS) tumors in children is among the most challenging problems in pediatric oncology. Many tumor types share overlapping microscopic features,…

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

  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 likelihood of urologic complications  increases with the complexity of the malformation.…

Mode of Respiratory Support at 36 Weeks Predicts Weaning Timeline for Infants With Severe Bronchopulmonary Dysplasia
 Mode of Respiratory Support at 36 Weeks Predicts Weaning Timeline for Infants With Severe Bronchopulmonary Dysplasia 1024 683 Lauren Dembeck
By: Lauren Dembeck
Premature baby receiving respiratory support through a nasal cannula in a neonatal intensive care unit, monitored and cared for by a health care provider.

Bronchopulmonary dysplasia (BPD) is the most common complication of preterm birth, and its incidence is rising due to advances in neonatal care that have improved survival for extremely premature infants. Respiratory support is a cornerstone of management for these patients, yet the expected timeline for weaning in those with moderate-to-severe (grade 2/3) BPD is not…

Study Explores Impact of Duffy Null Phenotype on Neutrophil Count and Hydroxyurea Dosing in Children With Sickle Cell Disease
 Study Explores Impact of Duffy Null Phenotype on Neutrophil Count and Hydroxyurea Dosing in Children With Sickle Cell Disease 1024 683 Lauren Dembeck
By: Lauren Dembeck

Children with sickle cell disease and the Duffy null phenotype may have lower measured neutrophil counts, but new research suggests that there are opportunities to optimize hydroxyurea regardless of Duffy null status. Sickle cell disease (SCD) primarily affects individuals of minority populations and can cause numerous morbidities, including vaso-occlusive pain, acute chest syndrome episodes, and…

High Rates of Readmission and Airway Complications Found in Tracheostomy-Dependent Infants With BPD
 High Rates of Readmission and Airway Complications Found in Tracheostomy-Dependent Infants With BPD 1024 575 Lauren Dembeck
By: Lauren Dembeck

While tracheostomy can be lifesaving for infants with severe bronchopulmonary dysplasia, new data highlights the risks and outcomes that families and providers must be prepared to face. Infants with bronchopulmonary dysplasia (BPD), the most common morbidity of prematurity, often require long-term respiratory support. Severe BPD can necessitate tracheostomy that is often paired with mechanical ventilation,…

Meet Our Expert: Albert Isaacs, MD, PhD, Neurosurgeon and Genomic Scientist
 Meet Our Expert: Albert Isaacs, MD, PhD, Neurosurgeon and Genomic Scientist 864 576 Lauren Dembeck
By: Lauren Dembeck
Neurosurgeon Albert Isaacs posed in the operating room, looking at the camera.

Albert Isaacs, MD, PhD, is a pediatric neurosurgeon at Nationwide Children’s Hospital and an assistant professor of Neurological Surgery at The Ohio State University College of Medicine. His research focuses on uncovering the molecular and immunologic drivers of neonatal hydrocephalus, with the goal of developing preventative treatments for at-risk infants. Dr. Isaacs’ extensive background and…

ACMG Issues Primer on Regulation of Laboratory-Developed Tests
 ACMG Issues Primer on Regulation of Laboratory-Developed Tests 1024 683 Lauren Dembeck
By: Lauren Dembeck

The new statement, co-authored by a Nationwide Children’s laboratory geneticist, outlines how lab-developed tests are regulated and offers patient-centered recommendations for modernizing oversight. Laboratory-developed tests (LDTs) are validated testing protocols developed by certified clinical laboratories for patient care and play an essential role in diagnosing and guiding treatment for many conditions that affect pediatric patients.…

New Trial Aims to Boost Kids’ Immune Systems to Fight Neuroblastoma
 New Trial Aims to Boost Kids’ Immune Systems to Fight Neuroblastoma 1024 558 Lauren Dembeck
By: Lauren Dembeck

A new cellular therapy approach could improve outcomes and ease the treatment burden for patients and families. Neuroblastoma is rare, with only 700 to 800 new cases diagnosed in the United States each year, but it is the most common extracranial solid tumor in children. Neuroblastoma typically occurs in children younger than 5 years, with…

Novel Viral Vector Manufacturing Approach Could Improve Safety of Gene Therapy
 Novel Viral Vector Manufacturing Approach Could Improve Safety of Gene Therapy 1024 683 Lauren Dembeck
By: Lauren Dembeck

New AAV proviral plasmid for vector manufacturing has potential to increase patient safety and make gene therapies more affordable, researchers say. Gene therapy is one of the most exciting frontiers in modern medicine, offering potential cures for previously devastating or fatal disorders, such as spinal muscular atrophy and aromatic L-amino acid decarboxylase deficiency. Adeno-associated virus…

Creation of Patient-Derived Induced Pluripotent Stem Cell Lines for Studying Rare Pediatric Genitourinary Disorders
 Creation of Patient-Derived Induced Pluripotent Stem Cell Lines for Studying Rare Pediatric Genitourinary Disorders 1024 683 Lauren Dembeck
By: Lauren Dembeck

Nationwide Children’s researchers create first-ever stem cell lines for rare genitourinary conditions. Induced pluripotent stem cells (iPSCs) have revolutionized medical research by providing powerful tools to study disease mechanisms, especially in rare genetic conditions. These versatile cells, derived directly from patients that have the condition, offer unprecedented opportunities to uncover the underlying genetic and cellular…

Eat, Sleep, Console: Reducing NICU Admissions of Infants With Prenatal Opioid Exposure
 Eat, Sleep, Console: Reducing NICU Admissions of Infants With Prenatal Opioid Exposure 1024 575 Lauren Dembeck
By: Lauren Dembeck

Focusing on non-pharmacological care, emphasizing mother-baby bonding, and natural infant behaviors may keep infants out of the NICU, but other barriers may limit implementation. Infants exposed to opioids in utero may experience withdrawal symptoms after birth, which can vary in severity depending on many factors, such as length of exposure and type of opioid. In…

Why Aren’t Pediatric Kidney Stone Patients Returning for Follow-up After Surgery?
 Why Aren’t Pediatric Kidney Stone Patients Returning for Follow-up After Surgery? 1024 732 Lauren Dembeck
By: Lauren Dembeck

Kidney stones in children are on the rise, and many of these children miss critical post-op follow-ups. What prevents them from returning, and how can we help? Since 2000, kidney stone cases in the U.S. pediatric population have risen by 6–10% annually, leading to a corresponding increase in surgical interventions. Alarmingly, nearly half of pediatric…

A Novel EEG Evaluation Method for Children With Infantile Spasms
 A Novel EEG Evaluation Method for Children With Infantile Spasms 1024 677 Lauren Dembeck
By: Lauren Dembeck

Clinician-researchers have developed a rapid training program to learn the BASED score, a novel EEG evaluation method. The easily accessible training program allows clinicians to reliably determine the response to treatment. Infantile epileptic spasms syndrome is a severe form of early childhood epilepsy. Onset typically occurs in infants 4 to 8 months old and is…

Establishing Meaningful Clinical Outcome Measures for Gastrointestinal Symptoms in Cystic Fibrosis
 Establishing Meaningful Clinical Outcome Measures for Gastrointestinal Symptoms in Cystic Fibrosis 1024 683 Lauren Dembeck
By: Lauren Dembeck

Understanding the minimal clinically important differences of PROMs is crucial in interpreting results in clinical trials. Patient-reported outcome measures (PROMs) are a set of standardized questions that directly assess the patient’s perspective of their symptoms and quality of life to better understand their experience with a disease or treatment. PROMs support patient-centered care and are…

Oncolytic Viroimmunotherapy Improved by Enhanced Cytokine Transgene Expression
 Oncolytic Viroimmunotherapy Improved by Enhanced Cytokine Transgene Expression 150 150 Lauren Dembeck
By: Lauren Dembeck

Researchers were able to suppress in vivo tumor growth with an oncolytic virus by enhancing the production of the cytokine interleukin-12 in the tumors. Malignant peripheral nerve sheath tumors (MPNSTs) are rare, aggressive sarcomas that exhibit resistance to non-surgical treatment and have high rates of metastasis and relapse. The prognosis for MPNSTs is poor, with…

Donor-Derived NK Cells Safe and Efficacious in Relapsed and Refractory AML
 Donor-Derived NK Cells Safe and Efficacious in Relapsed and Refractory AML 1024 649 Lauren Dembeck
By: Lauren Dembeck
artistic rendering of an NK cell on a faded blue background with other cells faintly visible in the background

Researchers have used their approach for growing large numbers of NK cells to allow repeated dosing for a more effective and less toxic treatment.   Outcomes for patients with relapsed and refractory acute myeloid leukemia (R/R AML) have not significantly improved over the last 30 years. For example, a recent study of patients treated with…

Practical Advice for Investigator-Initiated Trials
 Practical Advice for Investigator-Initiated Trials 1024 363 Lauren Dembeck
By: Lauren Dembeck

Investigator-initiated trials play a crucial role in advancing medical research and contributing to evidence-based medicine. These trials are initiated, designed and conducted by researchers, often physicians or scientists, rather than by pharmaceutical or biotechnology companies. They allow researchers to explore specific hypotheses, novel interventions or unique patient populations that may not be addressed in industry-sponsored…

Novel Immunotherapy Approach Enhances Immune Surveillance in Gliomas
 Novel Immunotherapy Approach Enhances Immune Surveillance in Gliomas 1024 575 Lauren Dembeck
By: Lauren Dembeck

Genetically engineered myeloid cells release interleukin-2, which helps the immune system overcome the immunosuppressive tumor microenvironment in gliomas.   Gliomas — tumors originating from the neuronal support (glial) cells of the central nervous system — are one of the leading causes of cancer-related death in adolescents and young adults. Approximately two-thirds of gliomas in these…

Neurorehabilitation for Children With Batten Disease
 Neurorehabilitation for Children With Batten Disease 1024 683 Lauren Dembeck
By: 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), and speech-language pathology therapy (SLP), can delay loss of function and…

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
By: 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, and require frequent monitoring of drug levels in the blood. Since…

The Endocrinologists’ Role in Caring for TPIAT Patients
 The Endocrinologists’ Role in Caring for TPIAT Patients 1024 500 Lauren Dembeck
By: Lauren Dembeck
illustrated cross section of islets from pancreas

Children with recurrent acute or chronic pancreatitis often suffer debilitating pain and may endure an impaired quality of life. They may be frequently admitted to the hospital, disrupting their daily routines and activities. Patients may undergo medical and endoscopic interventions for both diagnostic and therapeutic purposes and may be offered surgical interventions. Total pancreatectomy with…

Researchers Create Public Database of Nearly 40,000 Control Samples for Genetic Association Studies
 Researchers Create Public Database of Nearly 40,000 Control Samples for Genetic Association Studies 1024 575 Lauren Dembeck
By: Lauren Dembeck
conceptual art of DNA

Genetic association studies provide powerful means of discovering relationships between genetic variants and disease risk. However, these studies require enrolling very large numbers of individuals with the disease (cases) and healthy individuals (controls) as well as collecting genetic data for all of those participants — a time-consuming and expensive endeavor. “Sequencing DNA for a healthy…

Interactions Between Invading Tumor and Lung Cells Permit Metastatic Lung Colonization of Osteosarcoma
 Interactions Between Invading Tumor and Lung Cells Permit Metastatic Lung Colonization of Osteosarcoma 150 150 Lauren Dembeck
By: Lauren Dembeck

Survival signals elicited by lung tissue interactions promote osteosarcoma metastasis and represent a promising target for clinical trials in both human and canine patients.   Osteosarcoma, the most common primary tumor of bone, occurs predominantly in children, teens and young adults. Patient survival largely depends upon the presence or absence of metastasis. Within five years…

Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2
 Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2 150 150 Lauren Dembeck
By: Lauren Dembeck

Tuberous sclerosis complex (TSC) is a devastating genetic disease that affects nearly 1 in every 5,500 newborns and approximately 2 million people worldwide. The disease is characterized by the formation of non-malignant tumors throughout multiple organs, including the kidney, lungs, eyes, and heart, but predominantly the brain. It is typically diagnosed in infants and young…

GERD Less Likely Cause of Irritability and Back Arching in Infants
 GERD Less Likely Cause of Irritability and Back Arching in Infants 1024 683 Lauren Dembeck
By: Lauren Dembeck

In infants, frequent irritability and arching of the back can be concerning to parents and providers. These symptoms are often attributed to gastroesophageal reflux disease (GERD), a disease in which the lower esophageal sphincter does not close or opens at the wrong time. As a result, food and stomach acid can come back up into…

Tissue Engineered Trachea: State of the Research
 Tissue Engineered Trachea: State of the Research 150 150 Lauren Dembeck
By: Lauren Dembeck

The promise of tissue-engineered trachea grafts is moving closer to the clinic, as recent preclinical studies have shown successful implantation and neovascularization. Breathing is an essential biological function that provides our bodies with the oxygen necessary for survival. However, most of us rarely think about the biological structures that make it possible. The trachea, commonly…

Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors
 Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors 1024 683 Lauren Dembeck
By: Lauren Dembeck

The overall survival rates for children with childhood cancers is now over 80%; however, for children with relapsed and refractory tumors, the survival rates remain low. “For example, children with relapsed or refractory neuroblastoma have a five-year survival of less than ten percent,” explains Keri A. Streby, MD, director of the Neuroblastoma Program in the…

Racial Disparities in Health Care Use Among Medicaid-Covered Children With Congenital Heart Disease
 Racial Disparities in Health Care Use Among Medicaid-Covered Children With Congenital Heart Disease 1024 683 Lauren Dembeck
By: Lauren Dembeck

In the United States, congenital heart disease is the most common birth anomaly, with almost 40,000 newborns diagnosed each year. As these children grow, they are at increased risk of developing cardiovascular complications. Thus, ensuring they receive recommended cardiology care is essential to their long-term health and well-being. Racial disparities in health outcomes have been…

Coexistence of Different Telomere Maintenance Mechanisms in Pediatric High-Grade Glioma Tumors
 Coexistence of Different Telomere Maintenance Mechanisms in Pediatric High-Grade Glioma Tumors 1024 504 Lauren Dembeck
By: Lauren Dembeck

Due to within-tumor heterogeneity, telomere-based therapeutic interventions will likely need to target both known telomere maintenance mechanisms to prevent resistance and recurrence.   Normal differentiated somatic cells can divide only a limited number of times due to loss of the physical ends of their chromosomes, the telomeres, with each replication. When the telomeres reach their…

Recent Trends in the Diagnosis and Treatment of Anxiety in US Children and Adolescents
 Recent Trends in the Diagnosis and Treatment of Anxiety in US Children and Adolescents 1024 683 Lauren Dembeck
By: Lauren Dembeck

In 2021, the American Academy of Pediatrics, American Academy of Child and Adolescent Psychiatry and Children’s Hospital Association joined together to declare a national state of emergency in children’s mental health. The declaration was motivated by steadily increasing rates of childhood mental health concerns and suicide between 2010 and 2020. Among those concerns are anxiety…

Preemptive Stenting of the Left Pulmonary Artery During Surgery for Hypoplastic Left Heart Syndrome
 Preemptive Stenting of the Left Pulmonary Artery During Surgery for Hypoplastic Left Heart Syndrome 150 150 Lauren Dembeck
By: Lauren Dembeck

Perioperative stenting during the comprehensive stage 2 surgery does not negatively impact overall outcomes. However, Preemptive stenting has a survival advantage of babies relative to those who had reactionary stenting.   Each year, approximately 1,000 babies in the United States are born with one of the most common critical congenital heart defects—hypoplastic left heart syndrome.…

Targeted Muscle Reinnervation Prevents Neuroma and Phantom Limb Pain in Children After Amputation
 Targeted Muscle Reinnervation Prevents Neuroma and Phantom Limb Pain in Children After Amputation 1024 684 Lauren Dembeck
By: Lauren Dembeck

In certain cases, such as limb traumas and sarcomas, amputation may be chosen to optimize patients’ physical function. Persistent postamputation residual limb or “stump” pain and phantom limb pain can be debilitating and has been associated with prosthetic abandonment in patients who have undergone amputation. This pain is often secondary to symptomatic neuromas, tumor-like disorganized…

Kidney Support for Babies: Building a Comprehensive and Integrated Neonatal Kidney Support Therapy Program
 Kidney Support for Babies: Building a Comprehensive and Integrated Neonatal Kidney Support Therapy Program 1024 575 Lauren Dembeck
By: Lauren Dembeck
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

Kidney support therapy (KST), commonly referred to as dialysis, is a life-saving procedure used to manage complications associated with acute kidney injury and kidney failure, such as fluid overload and electrolyte imbalances, or to remove toxins, such as those in patients with inborn errors of metabolism. Most KST or dialysis machines are designed for adults…

Susceptibility to and Use of E-cigarettes and Marijuana Is Common Among Adolescents With Congenital Heart Disease
 Susceptibility to and Use of E-cigarettes and Marijuana Is Common Among Adolescents With Congenital Heart Disease 1024 575 Lauren Dembeck
By: Lauren Dembeck

Adolescents with congenital heart disease (CHD) are subject to disease-related stressors, such as attending medical appointments and undergoing medical procedures. They have elevated risk for cardiovascular and cognitive complications, which may be exacerbated by the use of e-cigarettes and marijuana. To inform prevention strategies for their long-term wellbeing, it is critical to understand patterns of…

Implementation of a COVID-19 Monoclonal Antibody Program
 Implementation of a COVID-19 Monoclonal Antibody Program 150 150 Lauren Dembeck
By: Lauren Dembeck

Researchers provide guidance on risk stratification and offer subspecialists and community practitioners a streamlined approach to treating patients at greater risk for severe COVID-19. The COVID-19 pandemic necessitated the development of therapeutic approaches to combat the SAR-CoV-2 virus. In late 2020, monoclonal antibody therapies were among the first COVID-19 therapeutics to receive emergency use authorization…

Improving the Diagnosis and Treatment of Iron Deficiency and Anemia in Children With Inflammatory Bowel Disease
 Improving the Diagnosis and Treatment of Iron Deficiency and Anemia in Children With Inflammatory Bowel Disease 150 150 Lauren Dembeck
By: Lauren Dembeck

Iron deficiency with and without anemia is under-recognized and under-treated in children with inflammatory bowel disease.   Anemia is the most common extr-aintestinal manifestation of inflammatory bowel disease (IBD), which includes conditions such as Crohn’s disease and ulcerative colitis, and iron deficiency is the most prevalent cause of anemia in children with IBD. Several factors…

The Role of a Children’s Hospital in Central Ohio’s Biotech Boom
 The Role of a Children’s Hospital in Central Ohio’s Biotech Boom 480 346 Lauren Dembeck and Natalie Wilson
By: Lauren Dembeck and Natalie Wilson

Nationwide Children’s Hospital is at the front of the pack among top National Institutes of Health (NIH) funded children’s hospitals when it comes to developing novel gene therapies and commercializing intellectual property. Achieving this level of commercial activity is a testament to the organization’s groundbreaking investments in infrastructure, unique approaches to engaging with industry and…

Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy
 Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy 1024 575 Lauren Dembeck
By: Lauren Dembeck

Duchenne muscular dystrophy (DMD) is a devastating progressive muscle-wasting disease that leads to loss of motor and cardiorespiratory function. The disease is caused by mutations in the DMD gene that result in the loss of expression of the dystrophin protein, which plays a critical role during contraction and relaxation of muscle. To date, the U.S.…

Substantial Variation in Fluid Balance Among Preterm Neonates
 Substantial Variation in Fluid Balance Among Preterm Neonates 1024 575 Lauren Dembeck
By: Lauren Dembeck
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

In neonates, approximately 75 to 90% of the body weight consists of body fluid. The amount varies with gestational age, with higher total body fluid percentages in extremely preterm infants, those born at 22 to 28 weeks of gestation. These extremely preterm infants have immature skin and kidneys and thus immature fluid homeostasis regulatory mechanisms,…

Study Identifies Multiple Cell Signaling Pathways for Calcification in Aortic Valve Disease Regulated by Nitric Oxide
 Study Identifies Multiple Cell Signaling Pathways for Calcification in Aortic Valve Disease Regulated by Nitric Oxide 1024 753 Lauren Dembeck
By: Lauren Dembeck

Exploring novel signaling mediators in calcific aortic valve disease may pave the way for the development of pharmacotherapies to treat this intractable condition during early stages of its progression.   Calcific aortic valve disease is a chronic inflammatory condition characterized by progressive fibrosis and calcification of the heart valves, which can cause narrowing of the…

Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis
 Exploring the Complex Immune Microenvironment of Rasmussen Encephalitis 1024 587 Lauren Dembeck
By: Lauren Dembeck

Researchers used a multi-omics approach to gain a deeper understanding of aberrant cellular signaling leading to progression of a rare childhood neurological disease.   Rasmussen encephalitis is a very rare childhood neurological disease characterized by chronic progressive inflammation of one hemisphere of the brain, leading to loss of function, neurodegeneration, and drug-resistant epilepsy. Researchers have…

Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions
 Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions 1024 575 Lauren Dembeck
By: Lauren Dembeck

The findings contribute to a scant body of literature on the changes in functional connection within the brain and neurocognitive outcomes in survivors of pediatric brain tumors. Pediatric brain tumors are the most common solid tumor diagnosis among children in the United States. With advances in treatment, the 5-year survival rate among these children and…

New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors
 New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors 1024 614 Lauren Dembeck
By: Lauren Dembeck
abstract art of magnifying glass over DNA strand

Collaboration among National Cancer Institute, Children’s Oncology Group and Nationwide Children’s Hospital will offer molecular characterization of childhood cancers. The National Institutes of Health’s National Cancer Institute (NCI), Children’s Oncology Group (COG), and Nationwide Children’s Hospital are poised to change the direction of pediatric cancer diagnosis and care through a new initiative that is the…

Brain Organoids Provide Novel Mechanistic Insight into Microcephaly Associated with AUTS2 Syndrome
 Brain Organoids Provide Novel Mechanistic Insight into Microcephaly Associated with AUTS2 Syndrome 873 552 Lauren Dembeck
By: Lauren Dembeck
Microscopy image of cerebral organoid sections

Researchers have functionally validated a genotype-phenotype relationship between a variant in the AUTS2 gene and neurodevelopmental deficits in AUTS2 syndrome using patient-derived brain organoids. Genetic variants in more than 100 genes, including the gene AUTS2, have been associated with autism spectrum disorders. However, the model systems used to study the mechanisms underlying how these genetic…

Discovering the Genetic Causes of Congenital Heart Defects
 Discovering the Genetic Causes of Congenital Heart Defects 1024 575 Lauren Dembeck
By: Lauren Dembeck

By studying the genomes of children and members of their family, researchers have discovered novel disease genes in families impacted by cardiac defects. Congenital heart disease is a leading cause of death in children under one year of age, and evidence supports a strong genetic component underlying this group of common developmental defects. However, historically,…

Do Symptom Scores and pH Impedance Correlate in Infants Treated for Gastroesophageal Reflux?
 Do Symptom Scores and pH Impedance Correlate in Infants Treated for Gastroesophageal Reflux? 1024 683 Lauren Dembeck
By: Lauren Dembeck

Gastroesophageal reflux (GER) — the passage of gastric contents into the esophagus — is a physiologic process that can be considered normal or abnormal depending on whether troublesome symptoms and/or complications also occur. In infants, it is difficult for clinicians to distinguish between GER and pathophysiologic GER disease (GERD). “Current diagnostic criteria for GERD are…