Pediatric neurology最新文献

{"title":"Gaps and Challenges in the Transition of Care in Neuromuscular Disorders With a Focus on Duchenne Muscular Dystrophy","authors":"Alessia Marcassoli MSc ,&nbsp;Nethmi Rajapakse BHSc ,&nbsp;Erika Guastafierro MSc ,&nbsp;Jo-Anne Petropoulos MIST ,&nbsp;Giulia Trucco MSc ,&nbsp;Angelica Mazzilli MSc ,&nbsp;Sebastian Friedrich MD ,&nbsp;Sunil Rodger MD ,&nbsp;Jana Willems MSc ,&nbsp;Delaney Ringer BHSc ,&nbsp;Julia Frei MD ,&nbsp;Anna Swain MSc ,&nbsp;Matilde Leonardi MD ,&nbsp;Nardo Nardocci MD ,&nbsp;Thorsten Langer MD ,&nbsp;Olaf Kraus De Camargo MD ,&nbsp;Jan Willelm Gorter MD, PhD ,&nbsp;Kinga Pozniak PhD ,&nbsp;Anne Fournier MD ,&nbsp;Rocio Gutierrez MSc ,&nbsp;Isabella Moroni MD","doi":"10.1016/j.pediatrneurol.2025.10.006","DOIUrl":"10.1016/j.pediatrneurol.2025.10.006","url":null,"abstract":"<div><h3>Background</h3><div>Transition from pediatric to adult care in neuromuscular disorders represents a great challenge for both patients and their families, and especially for patients with Duchenne Muscular Dystrophy. This review aims to investigate the transition experience from pediatric to adult care of patients and their families, considering different aspects of daily life.</div></div><div><h3>Methods</h3><div>Medline, Embase, PsycINFO, CINHAL, Web of Science, and SCOPUS electronic databases were searched to identify studies published from January 2000 to December 2024. From the initial 1955 research articles, 26 were finally included for qualitative synthesis. Studies reported information about transition process, activities of daily living, and relationships.</div></div><div><h3>Results</h3><div>Our results show the need, as expressed by patients and caregivers, for multidisciplinary collaboration between pediatric and adult care settings, proposing the adoption of an adolescent-centered approach that prompts patients to share their perspectives on specific health needs directly with caregivers. The findings underscore the importance of also considering the psychological and social aspects that can influence the transition path across different areas of life.</div></div><div><h3>Conclusions</h3><div>Without structured guidelines and indications, patients and families feel left alone during the transition process. Transition programs should consider all the life areas in which patients are involved, including strategies to improve engagement in social, educational, and professional settings.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"174 ","pages":"Pages 74-80"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145452549","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neuroblastic Tumor Causing Toe-Walking Without Mechanical Compression of the Spinal Cord or Nerve Roots","authors":"Thomas O'Neill MBBS ,&nbsp;Andrew J. Kornberg MBBS DipTA (ATAA), ACCAM ,&nbsp;Eppie M. Yiu MBBS, PhD","doi":"10.1016/j.pediatrneurol.2025.09.010","DOIUrl":"10.1016/j.pediatrneurol.2025.09.010","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 74-75"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145233175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unforeseen Positive Impacts of a Neonatal Encephalopathy Teleconsultation Program: Insights From Clinicians Practicing in a Rural State","authors":"Elizabeth Scharnetzki PhD ,&nbsp;Leah Marie Seften CCRP ,&nbsp;Clairette Kirezi ,&nbsp;Alexa Craig MD, MS, MSc","doi":"10.1016/j.pediatrneurol.2025.08.022","DOIUrl":"10.1016/j.pediatrneurol.2025.08.022","url":null,"abstract":"<div><h3>Background</h3><div>Virtual triaging using telemedicine has been associated with improved communication, efficiency and accessibility, and reduced costs of care delivery. The Maine Neonatal Encephalopathy Teleconsultation (Maine NET) Program was developed to provide these advantages to neonates in rural areas. In previous work, we discussed improved resource utilization, collaborative decision-making, communication, and continuity of care associated with Maine NET. Herein, we examine additional, unexpected themes related to the psychosocial impacts of Maine NET on participating clinicians.</div></div><div><h3>Methods</h3><div>We performed semistructured interviews from April 2018 to October 2022 with 16 clinicians representing all hospitals participating in Maine NET. We utilized hybrid thematic analysis to assess themes from transcribed interviews.</div></div><div><h3>Results</h3><div>Several themes of Maine NET were identified in this analysis, including reduced feelings of isolation, alleviation in intensity of time pressure, heightened stress and trauma related to caring for the newborns requiring resuscitation, increased equity and access to subspecialty care, and opportunities for education. One clinician stated that “…it's lonely at the top [when] you're in a rural community hospital running the show and it's always scary to make these big decisions…so having somebody share that … is super helpful for moral support.”</div></div><div><h3>Conclusions</h3><div>The Maine NET Program provided several unanticipated benefits to both patients and the clinicians caring for them. As rural states increasingly face health care workforce shortages, availability of teleconsultation may support physician recruitment and retention in low delivery volume hospitals as it improves care delivery and facilitates connection to pediatric specialists, reducing isolation and increasing opportunities for ongoing education.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 41-46"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145159600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Influence of Pediatric Long COVID Syndrome on Visual Perception and Neuropsychiatric Symptoms","authors":"Tzu-Yu Chen MS ,&nbsp;Yen-Ju Chu MD ,&nbsp;Chia-Jui Hsu MD ,&nbsp;Hsin-Pei Wang MD ,&nbsp;Lee-Chin Wong MD, PhD ,&nbsp;Wang-Tso Lee MD, PhD","doi":"10.1016/j.pediatrneurol.2025.08.018","DOIUrl":"10.1016/j.pediatrneurol.2025.08.018","url":null,"abstract":"<div><h3>Background</h3><div>Long COVID presents with a wide range of persistent symptoms and durations following coronavirus disease 2019 (COVID-19) infection. However, data on children and adolescents remain limited. This study aims to explore visual perception and neuropsychiatric symptoms in pediatric patients and examine their associations with brain volume differences.</div></div><div><h3>Methods</h3><div>A total of 60 participants, aged six to 18 years and confirmed COVID-19 antibody positive, were recruited five to eight months after infection. Owing to the diversity of symptoms, each symptom was assigned a weighted score from 0 to 3 based on its severity and relevance to brain function. Participants were then divided into two groups according to symptom severity. All participants underwent magnetic resonance imaging, and the Test of Visual Perceptual Skills-Fourth Edition (TVPS-4) was administered.</div></div><div><h3>Results</h3><div>The most common neuropsychiatric symptoms were headache or dizziness, along with attention and memory deficits, which persisted for up to six months. Gray matter volumes were significantly increased in the group with severe symptoms, particularly in subcortical and temporal regions. These brain volume differences showed significant correlations with both acute and chronic symptoms. In the TVPS-4 assessment, significant differences were observed in overall standard scores and in the Sequential Memory subtest between participants with visual-related symptoms and healthy control subjects.</div></div><div><h3>Conclusions</h3><div>Neuropsychiatric symptoms, impaired visual perception, and gray matter volume differences are evident in pediatric long COVID cases. The severity of neuropsychiatric symptoms during the acute phase may predict the degree of chronic-phase brain volume alterations. Longitudinal follow-up studies are essential to validate and expand upon these findings.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 22-29"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145159599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Retrospective Analysis of Current Clinical Utilization of Long-Term Video Electroencephalogram Monitoring at a Large Pediatric Tertiary Care Center","authors":"Phillip Sumardi BS ,&nbsp;Avni Sanghi DO ,&nbsp;Grace Gombolay MD ,&nbsp;Bryan Philbrook MD ,&nbsp;Ruba Al-Ramadhani MD ,&nbsp;Sonam Bhalla MD ,&nbsp;Jenny Lin MD","doi":"10.1016/j.pediatrneurol.2025.09.011","DOIUrl":"10.1016/j.pediatrneurol.2025.09.011","url":null,"abstract":"<div><h3>Background</h3><div>Long-term video electroencephalogram monitoring (LTVM) is critical to the detection and management of seizures. Prior work has shown that most seizures are detected within 24 hours of monitoring. In practice, many patients are monitored for longer especially those who are at higher risk for seizures. This study examines whether higher risk clinical characteristics affect seizure detection rate.</div></div><div><h3>Methods</h3><div>A retrospective chart review was conducted on 847 LTVM studies of patients aged 0-18 years between August 1, 2021, and January 31, 2022, for those who were acutely hospitalized. Demographic, clinical, and electroencephalogram data were reviewed and analyzed. Descriptive statistics were used to analyze data, comparing seizure yield between clinical indications and age subgroups.</div></div><div><h3>Results</h3><div>Median patient age was 1.5 years (interquartile range: 0.3-8.3 years). The most common indication for LTVM was seizure-like activity (51%, n = 429). The most common age group that utilized inpatient LTVM was infants (35%, n = 298). Seizures were detected in 22% (n = 189/847) of electroencephalograms. Median time to first detected seizure was 1.5 hours (interquartile range: 0.4-6.9 hours). Ninety-five percent of patients had seizures within 24 hours of LTVM initiation, regardless of age or indication. Comparison analysis revealed no statistical difference in overall seizure yield based on age or indication. High-risk subgroups of neonates, extracorporeal membrane oxygenation, and traumatic brain injury patients similarly did not have statistically different seizure yield on LTVM.</div></div><div><h3>Conclusions</h3><div>Almost all patients with seizures were detected within 24 hours regardless of age or clinical indication. These results indicate the need for standardization of LTVM resources.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 107-111"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145268862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recurrence Rate and Outcome of Varicella-Associated Childhood Stroke: A Nationwide Population-Based Study and Systematic Review","authors":"Nicol Dara Matoor MD ,&nbsp;Thomas Truelsen MD, PhD, DMSci ,&nbsp;Malene Landbo Boerresen MD, PhD ,&nbsp;Klaus Hansen MD, DMSci ,&nbsp;Alex Christian Yde Nielsen MD, PhD ,&nbsp;Christina Engel Hoei-Hansen MD, DMSci ,&nbsp;Julie Brix Bindslev MD","doi":"10.1016/j.pediatrneurol.2025.09.015","DOIUrl":"10.1016/j.pediatrneurol.2025.09.015","url":null,"abstract":"<div><h3>Background</h3><div>We performed a nationwide cohort study and systematic review to examine recurrence rate and clinical outcome in children with varicella-associated arterial ischemic stroke (AIS).</div></div><div><h3>Methods</h3><div>The cohort study included children with varicella-associated AIS (varicella &lt;12 months before stroke and/or confirmed varicella-zoster virus in cerebrospinal fluid) and was conducted in Denmark, a country without universal varicella-vaccination. The children were identified by searching the Danish National Patient Registry (2011-2020) and by registration of children assessed through a regional stroke triage-setup (2021-2023). Clinical data were extracted from medical records. The systematic review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. PubMed and Embase were searched for studies published from January 1, 2000, to June 26, 2023, reporting on recurrence rate in children (age &lt; 18 years) with varicella-associated AIS.</div></div><div><h3>Results</h3><div>In the cohort study, varicella-associated AIS was present in 32 of 181 (17.7%) children with AIS. Thirty-two children were treated with aspirin, 30 (93.8%) with antivirals, and 21 (65.6%) with steroids. Angiography descriptions were retrieved in 30 children and showed unifocal or multifocal stenoses in 12 (40%). Within median follow-up of 5.2 years, two (6.3%) children experienced stroke recurrence. Long-term deficits were observed in 21 (65.6%) children, but 29 (90.6%) achieved good functional outcomes. The systematic review yielded three eligible studies, including a total of 68 children. Reported recurrence rates ranged from 9.1 to 45.5% across studies.</div></div><div><h3>Conclusions</h3><div>This study demonstrates that varicella-zoster virus is an important AIS risk factor associated with a high risk of neurological deficits but a good functional outcome and low recurrence risk. Reported recurrence rates vary across the literature, likely reflecting small sample sizes but also differences in methodology and secondary prevention.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 132-141"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145302636","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Proof-of-Concept Study on Effectiveness of Nitrazepam in Resistant Infantile Epileptic Spasms Syndrome (NitRIS Trial)","authors":"Parth Lal MBBS ,&nbsp;Sandeep Negi PhD ,&nbsp;Kiran Prakash MD ,&nbsp;Dipika Bansal DM ,&nbsp;Naveen Sankhyan DM ,&nbsp;Jitendra Kumar Sahu DM","doi":"10.1016/j.pediatrneurol.2025.09.001","DOIUrl":"10.1016/j.pediatrneurol.2025.09.001","url":null,"abstract":"<div><h3>Background</h3><div>Globally, pediatric neurologists vary in their choice of antiseizure medications for children with infantile epileptic spasms syndrome (IESS) who are resistant to hormonal and vigabatrin therapy. This trial aimed to determine whether oral nitrazepam is more effective than oral topiramate in achieving complete cessation of epileptic spasms (ES) in such cases.</div></div><div><h3>Methods</h3><div>We conducted an open-label, randomized controlled trial with a superiority hypothesis and masked end point assessments. The primary effectiveness end point was complete and sustained cessation of ES for 4 weeks, assessed at 10 weeks post-randomization. Secondary end points included electroclinical remission, time to remission, quality of life (assessed using the Hindi version of the Quality of Life of the Infant scale), autonomic tone (measured via heart rate variability), and adverse events.</div></div><div><h3>Results</h3><div>Between June 2023 and December 2024, 80 children with resistant IESS were screened and 40 (29 boys, 11 girls) were randomized to receive oral nitrazepam (0.5-3 mg/kg/day; n = 20) or topiramate (2-12 mg/kg/day; n = 20). Complete cessation of ES was achieved in 55% with nitrazepam vs 15% with topiramate (<em>P</em> = 0.019) and electroclinical remission in 45% vs 10% (<em>P</em> = 0.031). Quality of life scores favored nitrazepam (<em>P</em> = 0.023). Parasympathetic heart rate variability was significantly higher in nitrazepam responders. Sedation was more common with nitrazepam and irritability with topiramate.</div></div><div><h3>Conclusions</h3><div>Nitrazepam showed superior short-term effectiveness over topiramate in resistant IESS, with better quality of life and autonomic stability. Further research is needed to assess long-term outcomes.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 66-73"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145222281","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Biological Sex and Body Mass Index Influence the Longitudinal Evolution of Adolescent and Young Adult Magnetic Resonance Imaging-Visible Perivascular Spaces","authors":"Erin A. Yamamoto MD ,&nbsp;Seiji Koike MAS ,&nbsp;Caitlyn Wong BS ,&nbsp;Laura E. Dennis BS ,&nbsp;Madison N. Luther BS ,&nbsp;Avery Scatena BA ,&nbsp;Seva Khambadkone MD, PhD ,&nbsp;Jeffrey J. Iliff PhD ,&nbsp;Miranda M. Lim MD, PhD ,&nbsp;Swati R. Levendovszky PhD ,&nbsp;Jonathan E. Elliott PhD ,&nbsp;Giuseppe Barisano MD, PhD ,&nbsp;Eva M. Müller-Oehring PhD ,&nbsp;Angelica M. Morales PhD ,&nbsp;Fiona C. Baker PhD ,&nbsp;Bonnie J. Nagel PhD ,&nbsp;Juan Piantino MD, MCR","doi":"10.1016/j.pediatrneurol.2025.08.021","DOIUrl":"10.1016/j.pediatrneurol.2025.08.021","url":null,"abstract":"<div><h3>Background</h3><div>An association recently emerged between magnetic resonance imaging (MRI)-visible perivascular spaces (MV-PVS) with intracerebral solute clearance and neuroinflammation, in adults. However, it is unknown how MV-PVS change throughout adolescence and what factors influence MV-PVS volume and morphology. This study assesses the temporal evolution of MV-PVS volume in adolescents and young adults, and secondarily evaluates the relationship between MV-PVS, age, sex, and body mass index (BMI).</div></div><div><h3>Methods</h3><div>This analysis included a 783 participant cohort from the longitudinal multicenter National Consortium on Alcohol and Neurodevelopment in Adolescence study that involved up to 6 imaging visits spanning 5 years. Healthy adolescents aged 12-21 years at study entry with at least two MRI scans were included. The primary outcome was mean MV-PVS volume (mm³/white matter cm³).</div></div><div><h3>Results</h3><div>On average, males had greater MV-PVS volume at all ages compared to females. A linear mixed-effect model for MV-PVS volume was performed. Mean BMI and increases in a person's BMI were associated with increases in MV-PVS volume over time. In females only, changes in BMI correlated with MV-PVS volume. One unit increase in BMI above a person's average BMI was associated with a 0.021 mm³/cm³ increase in MV-PVS volume (<em>P</em> &lt; 0.001).</div></div><div><h3>Conclusions</h3><div>This longitudinal study showed sex differences in MV-PVS features during adolescence and young adulthood. Importantly, we report that increases in BMI from a person's mean BMI are associated with increases in MV-PVS volume in females only. These findings suggest a potential link between MV-PVS, sex, and BMI that warrants future study.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 58-65"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145207140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From Crisis to Continuum: Redefining Survivorship in Neurometabolic Care","authors":"Bharatendu Chandra MBBS ,&nbsp;Madelyn Klemmensen BS ,&nbsp;Brian J. Shayota MD, MPH ,&nbsp;Andrea L. Gropman MD","doi":"10.1016/j.pediatrneurol.2025.08.017","DOIUrl":"10.1016/j.pediatrneurol.2025.08.017","url":null,"abstract":"<div><div>Advances in newborn screening, molecular diagnostics, and targeted therapies have markedly improved survival for individuals with inborn errors of metabolism (IEM), allowing many to live well into adulthood. While some may remain asymptomatic, their underlying metabolic vulnerabilities persist and can be unmasked by illness, stress, or other triggers. As a result, a growing population of adults with IEM now faces unique and evolving challenges related to long-term disease management, continuity of care, and quality of life. Despite these shifting demographics, there remains limited literature directly comparing pediatric and adult treatment outcomes, and emerging data suggest that therapeutic effectiveness in childhood may not always extend into adulthood. Survivorship in IEM spans a wide range of medical, neurodevelopmental, and psychosocial domains. These include maintaining metabolic stability, managing complex dietary needs, monitoring for progressive organ dysfunction, addressing neurocognitive outcomes, and navigating socioeconomic and mental health challenges. Compounding these issues are barriers such as limited access to specialized adult care, inconsistent insurance coverage for medical foods and therapies, and a lack of established adult-specific treatment protocols. This review explores the critical components of survivorship in IEM, including emerging therapies and the increasing importance of multidisciplinary care models. It underscores the necessity of well-coordinated transition programs that support adolescents and young adults as they shift from pediatric to adult healthcare systems. In addition, it highlights the need for expanded education and research efforts aimed at understanding and addressing the distinct and evolving needs of adults with IEM. By highlighting the lived experience, gaps in education, and complex care requirements of this growing patient population, this invited manuscript aims to inform tailored, lifespan-focused management strategies that reflect the realities of aging with a rare metabolic disorder.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 5-21"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150235","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reversal of Acute Liver Injury Following Delandistrogene Moxeparvovec Gene Therapy Using Sirolimus and Immunoglobulin","authors":"Serena M. Neumann APRN ,&nbsp;Jonathan H. Soslow MD ,&nbsp;William B. Burnette MD ,&nbsp;Rachel E. Harris DO ,&nbsp;Saeed Mohammad MD","doi":"10.1016/j.pediatrneurol.2025.09.003","DOIUrl":"10.1016/j.pediatrneurol.2025.09.003","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"173 ","pages":"Pages 76-78"},"PeriodicalIF":2.1,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145252253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}