A new gene therapy approach has been developed to deliver full-length versions of large genes, improving skeletal muscle function in patients with genetic disorders like dysferlinopathies. The strategy may hold new hope for treating limb girdle muscular dystrophy type 2B and other muscular dystrophies.
A new study found a 36% increase in track-related injuries among US youth between 1991 and 2008, with sprains and fractures being the most common injuries. The study suggests that injury prevention efforts should be tailored to different age groups and activities.
A new mouse model reveals that abnormal heart cell development during embryogenesis may be responsible for congenital heart disease. The study suggests that genetic mutations, such as the GATA4 mutation, can lead to underdevelopment of heart walls and septal defects.
Researchers at Nationwide Children's Hospital have created a new drug to target osteosarcoma, the most common bone tumor in children, using a modified version of Celebrex. The drug, 8A, selectively inhibits the STAT3 pathway, which is crucial for tumor formation and cancer progression.
A study published in the American Journal of Respiratory and Critical Care Medicine found that hot, humid air triggers an increase in airway resistance in patients with mild asthma, leading to coughing and airway constriction. Inhaling an asthma drug before exposure to hot, humid air prevented this reaction.
A new study found that the annual number of battery-related emergency department visits among children younger than 18 years of age more than doubled from 2,591 in 1990 to 5,525 in 2009. The majority of cases involved products not intended for young children, such as watches and remote controls.
A new study found that baby bottles accounted for 66% of injuries, followed by pacifiers at 20%, and sippy cups at 14%. The study suggests that children should be transitioned to regular cups at 12 months old and pacifier use limited after six months
A study found that amoxicillin-clavulanate triggers groups of propagated contractions within the small intestine, improving small bowel transit and influencing gut microbiome. The antibiotic may be effective in patients with alterations of duodenal phase III, chronic symptoms of intestinal pseudo-obstruction.
A study at Nationwide Children's Hospital has identified proteins in the blood that may indicate whether a mother's urinary tract infection affects fetal growth. The researchers found that infected mothers had higher levels of inflammatory protein IL-6, which correlated with lower birth weights and increased risk for prematurity.
A new approach to newborn screening for DMD uses a two-tier system with CK testing on dried blood spots and DNA testing, allowing for efficient and cost-effective diagnosis. This method has been validated in a pilot study of 37,749 newborn boys, detecting six cases of DMD gene mutations.
The Nationwide Children's Hospital will use the $35,000 grant to increase health education and information available to pregnant women on methadone treatment. The project aims to improve maternal and infant outcomes by strengthening relationships between healthcare providers and at-risk women.
A new study found that over 93,000 US children under 5 were treated in emergency departments for stair-related injuries from 1999-2008. The study recommends measures to prevent these injuries, including educating parents and modifying building codes.
A Nationwide Children's Hospital study found that inhaled nitric oxide treatment varies greatly across US NICUs, with no standardization in its use. Despite the lack of evidence supporting its routine use, iNO treatment remains common among premature infants.
A network approach improved outcomes for children with inflammatory bowel disease (IBD), increasing remission rates from 55% to 68%. The ImproveCareNow network implemented quality improvement efforts, leading to improvements in care processes and patient outcomes.
A study from Nationwide Children's Hospital found that excessive or insufficient weight gain during pregnancy does not affect a child's cognitive development. The research controlled for various confounding factors, including genetic and environmental influences, to separate the effect of gestational weight gain on cognition.
A Nationwide Children's Hospital study found that muscle regeneration may create an ideal environment for rhabdomyosarcoma to arise. The research uses mouse models of muscular dystrophy to investigate the growth of eRMS, a fast-growing and highly malignant tumor subtype.
A study by Nationwide Children's Hospital found that children with inflammatory bowel disease (IBD) experience more school difficulty than healthy children, mainly in regard to school absences. Internalizing problems are a significant predictor of absences and school-related quality of life.
A retrospective study by Nationwide Children's Hospital found significant weight loss and improvement in obesity-related diseases in morbidly obese adolescents after Roux-en-Y gastric bypass surgery. Four of five patients with high blood pressure resolved their condition within two years.
A nationwide study reveals that most young people who present to emergency departments with deliberate self-harm are discharged without receiving a mental health assessment. The study found that only 39% of patients discharged to the community received a mental health assessment while in the emergency department.
The St. Baldrick's Foundation has awarded a grant of $47,000 to the Biopathology Center at Nationwide Children's Hospital to enhance its digital pathology services. This funding will enable researchers to conduct more studies and enroll more children in clinical trials, which are their best hope for a cure.
Researchers discovered a transporter system in bacteria that protects itself from immune attack by transporting molecules meant to destroy it away from its inner membrane target. This mechanism helps bacteria survive in the host, but also presents an opportunity for developing novel antibiotics.
A new study examines changes in siblings one year after their brother or sister dies from cancer, finding positive and negative shifts in relationships and personality. Siblings reported greater maturity, compassion, and life priority changes compared to parents.
Dr. Gail Besner continues her research on HB-EGF for treating neonatal necrotizing enterocolitis (NEC), a leading cause of surgical death in premature infants. Recent data suggest that HB-EGF may improve intestinal function after NEC treatment.
A new feeding program at Nationwide Children's Hospital has improved oral feeding skills for premature infants, allowing them to be discharged earlier and without feeding tubes. The study found that 51% of infants were successfully feeding upon discharge and 84% at 1 year of age.
A new study by Nationwide Children's Hospital found that approximately 5,200 children were treated for window fall-related injuries each year, translating to 14 children per day. Children under 4 years old and those falling from heights over 3 stories or landing on hard surfaces are at increased risk.
Scientists at Nationwide Children's Hospital have developed a human-cell-derived model of ALS that accurately mimics the majority of cases. The new model reveals that astrocytes may be releasing toxins that contribute to motor neuron degeneration, highlighting the importance of replacing these cells as a potential therapy target.
A new study from Nationwide Children's Hospital helps clarify the definition of gastroesophageal reflux disease (GERD) in neonates. The research finds that symptoms can occur with acid, non-acid, gas, liquid, or mixed events, making treatment strategies based on modifying gastric acidity alone ineffective.
A new study found that portable pools are more dangerous than parents realize, with most fatalities and near-drownings occurring among young children. The Centers for Injury Research and Policy recommend multiple layers of protection to prevent submersion events.
Ependymoma, a devastating brain tumor in children, is chemo-resistant and incurable in up to 40 percent of cases. Researchers will investigate the role of the Ephb2 gene in ependymoma development.
A National Institutes of Health grant has been awarded to Nationwide Children's Hospital to develop a gene therapy for MPS IIIB, a fatal lysosomal storage disease. The therapy aims to deliver needed genes directly to the central nervous system by bypassing the blood-brain barrier.
A recent study at Nationwide Children's Hospital identified 11 patients with Self-Embedding Behavior (SEB), a form of self-injurious behavior involving foreign objects in soft tissue. The majority of patients had psychiatric disorders, histories of out-of-home placements, and suicidal ideations.
Nationwide Children's Hospital has been accepted into the Neonatal Research Network, a national organization dedicated to improving neonatal care. The center will contribute patient data to a registry of very low birth weight infants, providing valuable insights into premature infant health outcomes.
A new national study found that an estimated 5.25 million football-related injuries were treated in U.S. emergency departments between 1990 and 2007. The annual number of injuries increased by 27 percent, with nearly 2,000 children treated daily during football season.
Researchers at Nationwide Children's Hospital discovered two small molecule inhibitors that block STAT3, a protein linked to osteosarcoma. The study suggests these compounds may serve as a new, non-toxic treatment option.
A new national study found a significant increase in boxing injuries, including head and neck injuries, among participants 6 years of age and older. The study also revealed that concussions among younger boxers are a growing concern due to potential long-term neurological implications.
A nearly $2 million NIH grant will help Nationwide Children's Hospital investigate biomarkers linked to hemangioma development and outcome. The study aims to understand the role of oxidant production in hemangioma formation.
A new US study found an average of 9,500 injuries and over 100 deaths related to cribs, playpens and bassinets among children under 2 years old each year. The majority of injuries involved cribs, with the most common injury being soft-tissue injury.
A nationwide study has found a significant rise in running-related injuries among children and adolescents between 1994 and 2007. The research indicates that over 225,000 cases were treated in US emergency departments during this period.
Nationwide Children's Hospital has been awarded a $11.5 million contract extension to continue its role as a Biospecimen Core Resource for The Cancer Genome Atlas (TCGA). This award will enable the hospital to analyze tumor specimens, extract DNA and RNA, and distribute these samples to researchers nationwide.
Researchers developed a gene therapy strategy using parthenogenetic embryonic stem cells, which can differentiate into multiple tissue types and potentially reduce rejection problems. The approach shows promise for treating autosomal dominant diseases like Beta Thalassemia, tuberous sclerosis, or Huntington's disease
A nationwide study reveals that exertional heat-related injuries affect not only athletes but also everyday activities like yard work and home maintenance. People 40 years old and older are more susceptible to these injuries.
Researchers studying gene therapy for Duchenne muscular dystrophy discovered a natural immunity to dystrophin, complicating experimental therapies. The immune response triggered by T cells may target muscle cells, resembling autoimmunity.
Researchers at Nationwide Children's Hospital are developing a new method to distinguish between bacterial and viral infections in infants using RNA biosignatures. The five-year study aims to improve diagnosis and treatment for serious bacterial infections such as bacteremia and meningitis, which can be life-threatening.
A unique gastroenterology procedure, Double-Balloon Enteroscopy (DBE), has been successfully used to treat small intestinal diseases in children. DBE allowed doctors to diagnose and manage diseases such as Crohn's disease and Peutz-Jeghers Syndrome with improved symptoms.
Researchers identified molecular changes and right ventricular diastolic dysfunction in patients with restrictive ventricular septal defect, a seemingly benign congenital heart disease. Early detection and treatment may prevent future complications and improve outcomes for these patients.
A new study found a 70% increase in basketball-related traumatic brain injuries (TBIs) among children and adolescents between 1997 and 2007. The study, conducted by Nationwide Children's Hospital, also revealed that TBIs doubled for boys and tripled for girls during this time.
A $250,000 grant from the Sophia's Cure Foundation via the Pepsi Refresh Project will support SMA research and clinic development at Nationwide Children's Hospital. The grant will help Dr. Kaspar's team develop gene therapy methods for scalable production and purification to advance their program to clinical trials.
Scientists at Nationwide Children's Hospital have discovered a distinct blood transcriptional signature associated with active TB, which may help identify patients at risk of developing the disease. The study found that this signature was present in 10-20% of latent TB patients and correlated with disease extent in active TB patients.
The NIH grant will support research into novel treatments for muscular dystrophies, including gene replacement and repair methods.
A study by Nationwide Children's Hospital found an average of over 20,000 sledding-related injuries treated in US hospital emergency departments each year. The most common injuries were fractures and cuts/bruises, with a high risk of traumatic brain injury (TBI) during collisions.