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FDA approves first gene therapy treatment for Sanfilippo syndrome type A, developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital

The FDA approved FAYUVI, a gene therapy treatment for pediatric patients with Sanfilippo Syndrome Type A, following years of research at the Abigail Wexner Research Institute. The treatment, developed by Nationwide Children's Hospital, is the fourth gene therapy to gain FDA approval from the institution.

Hassenfeld Children’s Hospital at NYU Langone ranks No. 1 in New York for pediatric pulmonology and lung surgery

Hassenfeld Children's Hospital at NYU Langone has been ranked as the No. 1 children's hospital in New York State for pediatric pulmonology and lung surgery. The hospital offers advanced pulmonary care, including pediatric lung transplant expertise, and innovative treatments for complex respiratory and thoracic conditions.

Beyond the gut: Necrotizing enterocolitis drives neurodevelopmental impairment through gut-derived inflammation

A comprehensive review reveals that necrotizing enterocolitis fundamentally alters brain development in premature infants, leading to lasting cognitive, motor, and behavioral impairments. The study highlights the role of gut-derived inflammation in driving brain injury and identifies promising new avenues for intervention.

SourceZhejiang University·JournalWorld Journal of Pediatric Surgery·DateSep 15, 2026

University hospitals awarded historic $56.25 million HRSA grant to lead new Pediatric Prevention Network

The Pediatric Prevention Network, led by University Hospitals Cleveland Medical Center, aims to improve pediatric emergency preparedness and resilience nationwide. The network will develop evidence-based solutions to strengthen preparedness and improve outcomes for children, building on the expertise and experience of UH Rainbow Babies...

Feelings upon learning of pregnancy, child developmental delays, and the role of social support

A nationwide birth cohort study found that negative feelings about pregnancy are associated with child developmental delays, while social support reduces risks. Mothers who reported 'no specific feelings' when learning of their pregnancy had the strongest association with developmental delays, highlighting the importance of identifying...

SourceToyama Regional Center for the Japan Environment and Children's Study, University of Toyama·JournalJournal of Health Psychology·TypeObservational study·DateSep 9, 2026

Current blood pressure thresholds may miss critically injured children

A national study of over 230,000 pediatric trauma patients found that signs of hemodynamic instability may appear earlier than current standards indicate, highlighting the need for more accurate blood pressure thresholds. The study suggests that clinicians may need to recognize and respond to signs of circulatory instability at higher ...

SourceAmerican College of Surgeons·JournalJournal of the American College of Surgeons·TypeData/statistical analysis·DateSep 9, 2026

Bacterial trial fails to demonstrate early asthma prevention

A national trial found that treating young children with an oral bacterial extract did not reduce the risk of severe wheezing and respiratory illnesses that can precede asthma. The ORBEX trial, led by the University of Arizona, tested the preventive potential of OM-85, a treatment that had shown promise in earlier, smaller studies.

SourceUniversity of Arizona·JournalThe Lancet·TypeExperimental study·DateSep 8, 2026

Health warnings could reduce young people’s social media use, Stanford Medicine study finds

A Stanford Medicine study found that pop-up messages warning of social media harms, such as mental health consequences, can reduce young people's use and boost their knowledge of risks. The study, which tested 14 warnings, found that messages on overall mental health harms, depression, and anxiety resonated most with young people.

SourceStanford Medicine·JournalJAMA Health Forum·TypeSurvey·DateSep 4, 2026

Pediatric investigation study highlights gaps between autism screening and timely support

Researchers identified referral gaps, diagnostic waits, and service pressures that can delay action after early autism concerns emerge. The study suggests practical changes to improve coordination and timely support for families, including repeat surveillance, closed-loop referral tracking, and needs-based support.

SourcePediatric Investigation·JournalPediatric Investigation·TypeLiterature review·DateSep 3, 2026

Children’s Hospital of Philadelphia researchers find C. difficile colonization in infants reshapes gut development

A new study by CHOP researchers reveals that C. difficile colonization in infants can lead to a remodeling of the developing epithelium and a reshaping of the mucosal immune system, potentially increasing the risk of some viral infections in adulthood. Early life exposure to C. difficile may have profound effects on healthy development...

SourceChildren's Hospital of Philadelphia·JournalScience·TypeRandomized controlled/clinical trial·DateSep 3, 2026

UCLA Health expert weighs in on new treatment approach for children with short-limbed dwarfism

A phase 3 trial shows infigratinib treatment significantly improves growth in children with achondroplasia, a skeletal condition causing disproportionate short stature. The oral treatment option is particularly suitable for early intervention in infants, who are most likely to benefit from pharmacologic treatment.

SourceUniversity of California - Los Angeles Health Sciences·JournalNew England Journal of Medicine·TypeCommentary/editorial·DateSep 3, 2026

Time-dependent premature suture fusion in craniosynostosis

Researchers identified a critical postnatal window in which excessive FGFR2 signaling drives coronal craniosynostosis by reducing GLI1+ progenitors and promoting abnormal osteogenesis. Restoring suture patency or RA signaling rescues structural and neurocognitive abnormalities.

SourceEditorial Office of West China School of Stomatology, Sichuan University·JournalInternational Journal of Oral Science·TypeExperimental study·DateAug 28, 2026

Pediatric investigation study links early birth to malnutrition in cerebral palsy

A new study suggests that children with cerebral palsy born very preterm may need closer nutritional and rehabilitation monitoring due to higher risks of undernutrition and functional impairments. The study found that gestational age should be considered when assessing nutrition and rehabilitation needs in children with cerebral palsy.

SourcePediatric Investigation·JournalPediatric Investigation·TypeObservational study·DateAug 28, 2026

Study shows sharp increase in rate of unintentional ingestions of recreational drugs among young children since 2000

A new study reveals a 3,307% increase in recreational drug ingestions among young children from 2000 to 2024, with most ingestions involving cannabinoids. The ingestion rate of marijuana edibles increased by 2,349% from 2015 to 2024, driven by the rapid growth of marijuana edibles and state legalization of cannabis.

SourceNationwide Children's Hospital·JournalBMC Public Health·TypeData/statistical analysis·DateAug 27, 2026

National study led by Lurie Children’s finds physical exams alone may not reliably diagnose pneumonia in children

A national study led by Lurie Children's Hospital finds that doctors may not always agree on common signs of pneumonia, such as crackles and decreased breath sounds, when examining the same child. The study suggests that a physical exam alone may not be enough to confidently diagnose pneumonia in children, highlighting the need for bet...

Study led by Lurie Children’s supports earlier access to cochlear implants for children with hearing loss

A study by Lurie Children's Hospital of Chicago found that cochlear implants can significantly improve hearing and listening skills in children with severe hearing loss, even at younger ages. The study showed that most children experienced meaningful improvements within a year after receiving a cochlear implant.

Pennington Biomedical Research Center’s 2026 Childhood Obesity Conference: “Be the Reason Kids Greaux Healthy” to be held Sept. 24

The 2026 Childhood Obesity Conference, 'Be the Reason Kids Greaux Healthy,' will explore strategies for preventing and treating childhood obesity, bringing together healthcare professionals, researchers, educators, and community leaders. The conference will feature keynotes, sessions, and poster presentations on emerging topics in pedi...

Alcohol marketing exposure and youth drinking

A systematic review and meta-analysis revealed a strong association between alcohol marketing exposure and increased drinking outcomes among youth. The study's findings suggest that reducing alcohol marketing can help mitigate the risk of youth drinking, highlighting the need for targeted interventions to address this public health con...

SourceJAMA Network·JournalJAMA Pediatrics·DateAug 24, 2026

UC Davis-led initiative drives dramatic improvements in adolescent behavioral health screening nationwide

A nationwide quality improvement initiative led by UC Davis Health and the American Academy of Pediatrics increased screening and follow-up care for adolescent substance use and mental health concerns. Clinics implemented evidence-based strategies, including validated screening instruments, and reported significant improvements in pati...

SourceUniversity of California - Davis Health·JournalAmerican Journal of Medical Quality·TypeExperimental study·DateAug 24, 2026

Gene therapy breakthrough restores heart function

Researchers at Murdoch Children's Research Institute have made a breakthrough in gene therapy, restoring heart function in lab-grown patient heart tissue and mouse models with genetic heart disease. The therapy may also correct other genetic heart diseases, including those caused by the MYH7 gene and TTN truncating variants.

SourceMurdoch Childrens Research Institute·JournalNature Cardiovascular Research·TypeExperimental study·DateAug 19, 2026

Johns Hopkins Children’s Center study shows decision support guidelines safely reduce unnecessary testing for very sick children

A new study published in JAMA Network Open suggests that decision support guidelines can safely reduce unnecessary endotracheal aspirate culture testing for children with respiratory complications. The study, funded by the NIH and AHRQ, found a 16% decline in EAC culture testing among patients without signs of respiratory tract infection.

SourceJohns Hopkins Medicine·JournalJAMA Network Open·DateAug 18, 2026

Charles W.M. Roberts, MD, PhD, an internationally recognized physician-scientist, named president and CEO of St. Jude Children’s Research Hospital

Charles W.M. Roberts, a renowned physician-scientist, will lead St. Jude Children's Research Hospital in accelerating progress against childhood cancer and catastrophic diseases. With his expertise in cancer epigenetics and clinical research, Roberts aims to transform outcomes for children worldwide.

Experts lead European study into childhood survival inequalities

A major European study published in PLOS One found that children from disadvantaged backgrounds are more likely to die from congenital anomalies, with the risk increasing after infancy. The research highlights the influence of social and economic factors on health outcomes, particularly in countries with lower GDP per capita.

SourceSwansea University·JournalPLOS One·TypeData/statistical analysis·DateAug 14, 2026

Children’s Hospital of Philadelphia researchers identify key genetic differences between pediatric and adult forms of immune-mediated inflammatory diseases

Researchers from Children's Hospital of Philadelphia have found distinct genetic architecture in pediatric-onset rheumatic and inflammatory diseases compared to adults. The study identified 39 genome-wide significant variants associated with immune responses, including previously unreported variants.

SourceChildren's Hospital of Philadelphia·JournalAnnals of the Rheumatic Diseases·TypeExperimental study·DateAug 12, 2026

Largest real-world study finds early flu antiviral treatment reduces ICU admissions in hospitalized children

A nationwide study published in JAMA Pediatrics found that early antiviral treatment with oseltamivir reduced ICU admissions and shortened hospital stays for children hospitalized with influenza. The study of over 7,000 pediatric hospitalizations showed a 31% lower likelihood of ICU admission among treated children.

SourceUniversity of Colorado Anschutz·JournalJAMA Pediatrics·DateAug 10, 2026

Growth hormone therapy helps childhood stem cell transplant survivors grow taller, but responses vary widely

A Japan-wide study found that growth hormone therapy improves adult height in childhood stem cell transplant survivors with short stature. However, treatment responses were highly variable and influenced by transplant-related factors such as total body irradiation and chronic graft-versus-host disease.

SourceHiroshima University·JournalFrontiers in Endocrinology·TypeData/statistical analysis·DateAug 7, 2026

Type 1 diabetes incidence and prevalence in the US

A cross-sectional study examines state-level type 1 diabetes burden and pediatric endocrinology resource distribution in the US. The study found that type 1 diabetes incidence has increased over time, while pediatric endocrinology resources are not evenly distributed across states.

SourceJAMA Network·JournalJAMA Network Open·DateAug 7, 2026