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Monthly Archives :

March 2024

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…

Modulator Therapy Improves Chronic Sinus Disease in Individuals with Cystic Fibrosis
 Modulator Therapy Improves Chronic Sinus Disease in Individuals with Cystic Fibrosis 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Following 1 year of therapy, patients showed improved sinus CT metrics and clinical outcomes. Over the last decade, the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs has revolutionized the treatment of cystic fibrosis (CF). The most widely used modulator is the triple combination elexacaftor-tezacaftor-ivacaftor (ETI), which is approved for people with CF…

What Pulmonologists Need to Know About Respiratory Insufficiency in Neuromuscular Disease
 What Pulmonologists Need to Know About Respiratory Insufficiency in Neuromuscular Disease 600 400 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

The RIND Study Group has released new consensus criteria to proactively identify hypoventilation in children with neuromuscular diseases. Recently released consensus criteria from an expert panel of pediatric pulmonologists and sleep specialists define and provide diagnosis guidance surrounding respiratory insufficiency in neuromuscular disease (RIND). The results from the 15-member RIND Study Group’s Delphi study, published…

Featured Researcher – Elaine Mardis, PhD
 Featured Researcher – Elaine Mardis, PhD 150 150 Alaina Doklovic
By: Alaina Doklovic

Elaine Mardis, PhD is co-Executive Director of the Steve and Cindy Rasmussen Institute for Genomic Medicine (IGM) at Nationwide Children’s Hospital and holds the Rasmussen Nationwide Foundation Endowed Chair in Genomic Medicine. She is also a Professor of Pediatrics at The Ohio State University College of Medicine. Dr. Mardis is an internationally recognized expert in…

At the Heart of a First R01 Grant
 At the Heart of a First R01 Grant 150 150 Alaina Doklovic
By: Alaina Doklovic

Allison Bradbury, MS, PhD, is a principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital and an assistant professor in the Department of Pediatrics at The Ohio State University. She recently received her first R01 grant to develop novel therapeutic strategies for tubulin folding cofactor D (TBCD)-related developmental and epileptic encephalopathy. This…

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

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

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

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

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

Recent study evaluated the safety and efficacy of using magnetically controlled intramedullary nails in patients with programmable implantable devices, shedding light on a previously unexplored area of orthopedic surgery. In a pivotal study published in the Strategies in Trauma and Limb Reconstruction, led by Christopher A. Iobst, MD, a pediatric orthopedic surgeon at Nationwide Children’s…

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

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

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

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

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

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

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

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