Researchers have identified patterns of spinal curvature in younger children that may develop into scoliosis by adolescence. The study suggests that the shape of a person's sagittal profile can be a leading cause of scoliosis, and may eventually guide early interventions for children at risk.
A new group cognitive-behavioral therapy program is being used to help parents of children with Avoidant/Restrictive Food Intake Disorder (ARFID) improve their child's mealtime behaviors. The program, known as the Picky Eaters Clinic, has reported life-changing improvements in parents and their children.
Stephan Grupp, a leading pediatric oncologist, has been elected to the National Academy of Medicine for his groundbreaking work in cell and gene therapy. He is recognized for developing an entirely novel therapy for acute lymphoblastic leukemia and leading the first global engineered cell therapy trial.
Beverly Davidson, a leading neuroscientist and gene therapy expert, has been elected to the National Academy of Medicine. Her research focuses on understanding neurodegeneration in inherited brain disorders and developing novel molecular medicines.
Researchers have identified a new neurodevelopmental syndrome caused by mutations in the NKAP gene, leading to developmental delay, intellectual disability, behavioral abnormalities, and musculoskeletal problems. The condition affects only males and is characterized by Marfanoid traits and cognitive impairment.
Researchers found the widely used M-CHAT/F screening tool is less accurate in real-world settings than shown in previous studies. The study also revealed disparities in detecting early autism symptoms in minority, urban, and low-income children. Despite these findings, experts recommend continuing universal screening for all toddlers.
Researchers used state-of-the-art brain imaging techniques to study nonverbal children with autism, finding delayed responses to simple auditory tones. The study suggests that measuring brain activity in the auditory cortex could serve as important objective markers for response to sound.
Researchers found that female athletes had longer recovery trajectories and experienced more symptoms than males due to delayed presentation to specialist care. The study suggests that inequities in medical and athletic trainer coverage on the sidelines may contribute to these delays.
A study has identified TCF4 as a master regulator of schizophrenia during early human brain development. The gene is believed to play a crucial role in the disease's underlying genomic biology. Researchers hope that this discovery will pave the way for new treatments and precision medicine approaches.
Researchers discovered a receptor on brain cells that promotes resilience to stress and may be a useful biomarker for post-traumatic stress disorder (PTSD). The sphingosine-1-phosphate receptor 3 (S1PR3) protein was found to be associated with reduced symptoms of PTSD in human patients.
A large-scale study has identified a robust new signal and fine-mapped previously reported genetic variants associated with childhood obesity. The findings suggest that genetic influences on obesity operate across the lifespan and provide valuable insights into potential treatments.
Researchers from Children's Hospital of Philadelphia found that brief parent-targeted interventions can increase parent-teen communication about sexual and alcohol-related behavior. Parent-reported frequency did not differ by group.
A randomized controlled trial found that ultrasound-guided IV line placement increased first-attempt success rates to 85.4% compared to traditional methods at 45.8%. Children's Hospital of Philadelphia researchers report a significant reduction in needle sticks and improved patient satisfaction.
Children's Hospital of Philadelphia researchers have developed a new AAV vector screening method that captures the full range of gene expression patterns caused by AAV vectors. This innovative technique is expected to significantly advance the field of gene therapy by providing a more sensitive approach to detecting gene transfer sites.
Researchers at Children's Hospital of Philadelphia identified a gene mutation responsible for a complex defect in a young boy's body. They harnessed this knowledge to develop a novel treatment that dramatically improved the boy's condition, reshaping his abnormal lymphatic vessels into a more normal anatomy and function.
Researchers developed an algorithm to identify pediatric oncology patients in EHR data, narrowing down patient cohorts for clinical studies. The tool improved accuracy and efficiency in patient selection, paving the way for more effective research on childhood cancers.
A study by Children's Hospital of Philadelphia found that autistic adolescents need the support of their parents or guardians to prioritize independence and develop life skills before learning to drive. Driving instructors emphasize the importance of parent-supervised practice, individualized training, and prioritizing independent living.
A new study from Children's Hospital of Philadelphia finds that a skin patch may be useful in treating children with eosinophilic esophagitis triggered by milk. Nearly half of the 20 children who wore the Viaskin Milk patch saw improvements in their symptoms and normalization of biopsies after 11 months.
A study by Children's Hospital of Philadelphia found that teen drivers with ADHD are at higher risk of crashes and traffic violations due to risky driving behaviors. The study analyzed records of 14,936 adolescents and identified 1,769 with childhood-diagnosed ADHD.
A team of researchers discovered a new gene linked to severe childhood epilepsy by analyzing phenotypes and genomics data of patients with similar clinical presentations. The study used a novel computational method to identify the AP2M1 gene variant, which regulates excitability and inhibition in brain cells.
A study found that parents of children with cancer frequently search for health-related information, including ways to support their child and logistical issues. The study suggests that healthcare providers should consider these day-to-day concerns when offering family education and support.
A study found that transplanting gut bacteria from vulnerable rats to non-stressed rats altered depressive-type behaviors, while also causing brain inflammation. The researchers believe this may lead to probiotic treatments for human psychiatric disorders like depression.
The Pennsylvania Pediatric Medical Device Consortium has awarded four companies with $50,000 each to develop innovative medical devices for children. Devices include a rapid platelet monitoring system and tracheostomy tube dislodgement alarm, which aim to improve care for newborns and pediatric patients.
Researchers at Children's Hospital of Philadelphia present two innovative studies on virtual reality technology for training children with autism to interact with police, and a routine developmental screening method to identify developmental deceleration. The studies show promise in improving early detection of autism spectrum disorder.
A new study from Children's Hospital of Philadelphia found that autistic girls use more cognitive process words than boys, despite similar levels of autism severity. This suggests the need for sex-informed screening and diagnostic methods to identify girls with autism at an earlier age.
Using CRISPR gene editing, researchers have successfully treated lethal lung diseases in mice by introducing genetic changes during fetal development. The study shows promise for developing new therapies for congenital lung diseases, such as surfactant protein deficiency and cystic fibrosis.
Researchers found threshold levels of circulating factor VIIa that prevented bleeding episodes in animal models, potentially leading to a new preventive treatment for patients with hemophilia who develop antibodies to standard maintenance therapy.
A new study published in the Journal of Pediatric Surgery found that post-operative success rates for pediatric thyroid patients correlate with institution patient volume. The study showed extremely low complication rates, underscoring the importance of high-volume centers with specialized pediatric surgeons.
The Pediatric Cell Atlas will create a high-resolution data reference for child development, benchmarking healthy and abnormal tissues at the level of single cells. This will provide a standard tool for researchers to better understand childhood illnesses and develop precision treatments.
A large multicenter trial found that standard intermittent positive pressure ventilation did not reduce the risk of bronchopulmonary dysplasia (BPD) or death at 36 weeks postmenstrual age for extremely preterm infants. In contrast, sustained inflations did not appear beneficial in reducing BPD risk and mortality in this vulnerable popu...
Researchers have developed a new analytical tool that uses deep learning to analyze RNA splicing patterns from modestly covered RNA sequencing data sets. This tool, DARTS, enables scientists to discover disease biomarkers and therapeutic targets more efficiently.
A new study reveals that patients and their families have borne an increased financial burden for growth hormone treatment since its FDA approval in 2003. The cost burden has risen by 234% due to increased copayments, deductibles, and insurance restrictions.
Researchers have identified two new genes, ING3 and EPDR1, that affect bone-forming cells and may lead to stronger bone mineral density and fracture prevention. The study's innovative use of three-dimensional genomic geography analysis could also aid in the discovery of new treatments for other genetic diseases.
A preclinical therapy attached to an antibody targeting the ALK protein shows effectiveness against most neuroblastoma cells, killing cancer cells with minimal harm to healthy tissues. This approach could lead to new treatment options for aggressive forms of neuroblastoma and other high-mortality childhood cancers.
Researchers identified over 30 variations in the MT-ATP6 gene with diverse clinical symptoms and biochemical features. The study highlights the need for more precise clinical tests to determine treatment options for patients affected by mitochondrial disorders.
A study found that automated programs can identify sepsis in sick infants hours before clinicians recognize it, offering potential for improved clinical outcomes. Machine-learning models analyzed patient data from electronic health records to predict which infants had sepsis.
Researchers found therapeutic potential in a rare kidney disease treatment for some mitochondrial disorders, improving mitochondrial metabolism and reducing oxidative stress. However, dosages must be carefully controlled due to narrow therapeutic windows.
New studies provide insight into the struggles of children on the autism spectrum with co-occurring ADHD symptoms, highlighting difficulties with adaptive behavior and brain connectivity. Researchers found a strong relationship between ADHD symptoms and adaptive behavior scores in both home and school settings.
Researchers have developed a new map of newborn babies' brains that could provide a reference tool for studying typical brain development and neurological disorders. The study used noninvasive MRI scans to reveal complex brain architecture, offering potential biomarkers for autism spectrum disorder at birth.
Researchers at Children's Hospital of Philadelphia have discovered that specialized lung cells emerge early in fetal development, contributing to prematurity-related lung injuries. The study reveals the role of signaling pathways in shaping these cells and paves the way for future therapeutic interventions.
Research at Children's Hospital of Philadelphia found opioid prescribing for pediatric patients declined in shorter treatment durations and lower doses between 2013 and 2017. The rate of prescribing remained consistent, however, with a bias towards females, ethnic minorities, and public insurance patients.
Researchers report on successful separation surgery using advanced technologies and multidisciplinary team, showcasing improved outcomes for infants with craniopagus conjoined twins. The Delaney twins are thriving two-year-olds, exceeding expectations with their development and growth after the 11-hour separation surgery.
The Pennsylvania Pediatric Medical Device Consortium has partnered with University of Pittsburgh programs McGowan Institute for Regenerative Medicine and sciVelo to develop medical devices for children. The consortium, based at Children's Hospital of Philadelphia, has received a five-year $5 million grant renewal from the FDA.
CHOP surgeons found that opioids were often overprescribed for children's elbow fractures, with patients using less than 25% of the prescribed amount. Most patients stopped taking opioid medication after a short period, with some opting for over-the-counter pain relievers instead.
A recent study found that over 40% of youth exhibit obsessive or compulsive behaviors, which can be a red flag for serious psychiatric conditions. Children who experience bad thoughts and intrusive images are more likely to develop depression and suicidal tendencies.
Researchers evaluated a video game-based treatment for children with autism spectrum disorder (ASD) and co-occurring attention/deficit-hyperactivity disorder (ADHD). The study found that children engaged well with the treatment, showed improved attention, and reported high satisfaction rates.
Children's Hospital of Philadelphia celebrates European Commission approval of LUXTURNA, a one-time gene therapy treating vision loss due to inherited retinal dystrophy. The therapy has received regulatory approval in both the US and EU, offering new hope for patients with progressive blindness.
Children's Hospital of Philadelphia researchers present updated efficacy and safety data on Kymriah, a personalized CAR T-cell gene immunotherapy for aggressive blood cancers. The therapy achieved an 82% remission rate within three months in patients with relapsed or refractory ALL.
Researchers at Children's Hospital of Philadelphia identify the USMG5 gene mutation as a common cause of Leigh Syndrome, a mitochondrial disease causing progressive muscle weakness and energy deficits. The study provides long-awaited answers for parents affected by the condition.
A new genetic cause for a severe childhood epilepsy syndrome has been discovered, with spontaneous mutations in the CACNA1E gene disrupting calcium flow in brain cells. The study found potential medical treatments, including anti-seizure medications that target the affected channel.