Neurology最新文献

{"title":"Global Changes in Ischemic Stroke Burden Attributable to Ambient PM2.5: Trends From 1990 to 2020 and Projections to 2050.","authors":"Yisen Yang,Kai Li,Jing Xu,Meiduo Zhao,Qun Xu","doi":"10.1212/wnl.0000000000213692","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213692","url":null,"abstract":"BACKGROUND AND OBJECTIVESIschemic stroke (IS) is a leading cause of disability and mortality worldwide, with ambient fine particulate matter (PM2.5) exposure being a significant modifiable risk factor. While PM2.5 concentrations have declined in some regions, global assessments examining how these changes affect the IS burden remain limited. The aim of this study was to quantify changes in IS burden attributable to PM2.5 from 1990 to 2020 and project future trends to 2050.METHODSWe used data from the 2021 Global Burden of Disease study, including population estimates, IS incidence rates, and PM2.5 concentrations, for 204 countries and World Bank regions. The Environmental Benefits Mapping and Analysis Program algorithm was applied to estimate IS cases attributable to PM2.5. Future projections were calculated using an autoregressive integrated moving average model.RESULTSBetween 1990 and 2020, global PM2.5 concentrations decreased by 8.18 μg/m3. This reduction was associated with approximately 920,245 avoided IS cases, equivalent to 12.11% of the global IS incidence in 2020. The East Asia and Pacific region experienced the greatest benefit, with 699,218 IS cases avoided (19.09% of the region's IS incidence in 2020). By 2050, PM2.5 concentrations are projected to decline by 33.64 μg/m3 relative to 1990, potentially preventing an additional 6,004,854 IS cases. However, significant disparities persist, particularly in low-income regions where PM2.5 exposure and limited health care infrastructure continue to pose challenges.DISCUSSIONOur findings highlight the substantial public health benefits of PM2.5 reductions in mitigating the IS burden. While high-income regions have seen substantial gains due to stringent air quality regulations, low-income regions remain disproportionately affected. Addressing these disparities requires targeted pollution control policies and equitable health care resource allocation. Sustained efforts in air quality management are critical to reducing the global IS burden and improving health outcomes, particularly in vulnerable populations.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"28 1","pages":"e213692"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065878","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reader Response: Association Between Time to Treatment With Endovascular Thrombectomy and Home-Time After Acute Ischemic Stroke.","authors":"Kamil Cameron,James Ernest Siegler","doi":"10.1212/wnl.0000000000209945","DOIUrl":"https://doi.org/10.1212/wnl.0000000000209945","url":null,"abstract":"","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"43 1","pages":"e209945"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065880","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Olfaction and Plasma Biomarkers of Alzheimer Disease and Neurodegeneration in the Atherosclerosis Risk in Communities Study.","authors":"Srishti Shrestha,Xiaoqian Zhu,Michael E Griswold,Priya Palta,Kevin J Sullivan,Honglei Chen,Andrea Lauren Christman Schneider,Abhay Moghekar,Megan L Grove,Bharat Thyagarajan,James Russell Pike,Rebecca F Gottesman,B Gwen Windham,Thomas H Mosley,Jennifer A Deal,Vidyulata Kamath","doi":"10.1212/wnl.0000000000213706","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213706","url":null,"abstract":"BACKGROUND AND OBJECTIVESInvestigation of olfactory impairment, an early manifestation of Alzheimer disease (AD), in relation to plasma biomarkers of AD and neurodegeneration could provide insights into the disease's pathophysiology. Because few such studies based on large, diverse, community-based populations exist, we investigated associations of odor identification ability with plasma biomarkers of AD and other neurodegenerative pathologies in community-dwelling Black and White older adults.METHODSThis cross-sectional investigation included participants from the Atherosclerosis Risk in Communities study who attended visit 5 (2011-2013) and who completed olfactory testing and brain MRI examinations and had plasma biomarkers measured (namely, amyloid-beta [Aβ]42/Aβ40 ratio, phosphorylated-tau at threonine-181 [p-tau181], p-tau181/Aβ42 ratio, glial fibrillary acidic protein [GFAP], and neurofilament light chain [NfL]). Odor identification ability was measured by the 12-item Sniffin' Sticks test. Separate linear regression models were used to examine the association of continuous olfaction score and olfaction categories (anosmia: score 6; hyposmia: 7-8; moderate-normal: 9-10; good-normal: 11-12) with each biomarker (all were log-transformed), adjusting for sociodemographic and cardiovascular factors, head injury, APOE-ε4 status, and estimated glomerular filtration rate. We further examined whether any observed associations are explained by total and regional brain volumes.RESULTSAmong 1,545 participants (age: 76 ± 5 years, 60% women, 27% self-reported Black participants), the mean olfaction score was 9.2 ± 2.3; 14% had anosmia. Consistent with our hypotheses, poorer olfactory scores were associated with higher plasma p-tau181 (β per 1-unit lower score: 0.026 [95% CI 0.012-0.040]), p-tau181/Aβ42 (β: 0.027 [95% CI 0.011- 0.044]), GFAP (β: 0.024 [95% CI 0.009-0.040]), and NfL (β: 0.034 [95% CI 0.019-0.050] and lower Aβ42/Aβ40 ratio (β: -0.007 [95% CI -0.015 to 0.000]). Likewise, compared with good-normal olfaction, anosmia showed associations with all biomarker levels indicative of greater neuropathology (e.g., β for plasma p-tau181/Aβ42: 0.235 [95% CI 0.113-0.358] and β for plasma NfL: 0.210 [95% CI 0.102-0.317]), although the association with Aβ42/Aβ40 ratio was not statistically significant (β: -0.054 [95% CI -0.108 to 0.001]). These biomarkers were not significantly associated with hyposmia or moderate-normal olfaction. Smaller medial-temporal lobe volumes partly explained olfaction's link with plasma p-tau181, p-tau181/Aβ42, GFAP, and NfL.DISCUSSIONOur findings suggest that poor olfaction is associated with multiple AD-related and other neurodegenerative processes. Future studies should investigate how longitudinal changes in both olfaction and biomarkers relate to each other.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"124 1","pages":"e213706"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Teaching Video NeuroImage: An Unusual Etiology of Focal Epilepsy With Sensorimotor Reflex Seizures.","authors":"Elena Tartara,Angela Mariaelena Alicino,Paolo Vitali,Sabrina Dispenza,Anna Pichiecchio,Carlo Andrea Galimberti","doi":"10.1212/wnl.0000000000213704","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213704","url":null,"abstract":"","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"88 1","pages":"e213704"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065882","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dementia Risk and Obesity: Does Type of Fat Matter?","authors":"Nikolaos Scarmeas,Mary Yannakoulia","doi":"10.1212/wnl.0000000000213762","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213762","url":null,"abstract":"","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"201 1","pages":"e213762"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Associations Between Changes in Levels of Phosphorylated Tau and Severity of Cognitive Impairment in Early Alzheimer Disease.","authors":"Fernando Gonzalez-Ortiz,Bjørn-Eivind Kirsebom,Yara Yakoub,Julia K Gundersen,Lene Pålhaugen,Knut Waterloo,Per Selnes,Jonas Alexander Jarholm,Berglind Gísladóttir,Arvid Rongve,Ragnhild Eide Skogseth,Geir Bråthen,Dag Aarsland,Michael Turton,Peter Harrison,Henrik Zetterberg,Sylvia Villeneuve,Tormod Fladby,Kaj Blennow","doi":"10.1212/wnl.0000000000213676","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213676","url":null,"abstract":"BACKGROUND AND OBJECTIVESAligning biomarker evidence with clinical presentation in early Alzheimer disease (AD) is essential for improving diagnosis, prognosis, and interventions. This study evaluates the relationship between cognitive impairment, future decline, and phosphorylated tau levels in plasma and CSF in predementia AD.METHODSThis longitudinal observational study included predementia cases and controls from 2 independent cohorts: the Norwegian Dementia Disease Initiation (DDI) and Canadian Pre-Symptomatic Evaluation of Experimental or Novel Treatments for Alzheimer's Disease (PREVENT-AD). In DDI, cognitively normal (CN) and mild cognitive impairment (MCI) cases were classified using CSF Aβ42/40 ratio (A) and p-tau181 (T), whereas classification in PREVENT-AD (A) was based on amyloid PET scans. In DDI, we assessed CSF-plasma correlations for p-tau181, p-tau217, and p-tau231. Diagnostic accuracies were evaluated through receiver operating characteristic analyses. Linear mixed models evaluated p-tau associations with future memory decline. Between-group differences in plasma p-tau217 were assessed in both cohorts.RESULTSIn DDI (n = 431), participants were classified as CN A-/T- (n = 169), A+/T- (CN = 26, MCI = 24), A+/T+ (CN = 40, MCI = 105), and A-/T+ (CN = 34, MCI = 33), with a mean age of 64.1 years and 55.9% female. In PREVENT-AD (n = 190), participants were categorized as CN A- (n = 118), CN A+ (n = 49), and MCI A+ (n = 21), with a mean age of 67.8 years and 72.6% female. In DDI, plasma p-tau217 showed high accuracy in identifying A+ participants (areas under the curve [AUC]: 0.85) and a moderate correlation with CSF p-tau217 (rho = 0.65, p < 0.001). Diagnostic accuracy of plasma p-tau217 was greater in MCI A+ (AUC: 0.89) than in CN A+ (AUC: 0.79, p < 0.05) and in A+/T+ (AUC: 0.88) vs A+/T- (AUC: 0.78, p < 0.05). p-Tau181 and p-tau231 had weaker CSF-plasma correlations (rho = 0.47 and rho = 0.32, p < 0.001) and were less associated with cognitive status in A+ individuals. Higher plasma p-tau217 in A+ MCI vs A+ CN individuals (p < 0.001) was confirmed in PREVENT-AD. All CSF p-tau markers, but only plasma p-tau217, were associated with future memory decline (β = 0.05, p < 0.05).DISCUSSIONOur findings suggest that, unlike p-tau181 and p-tau231, plasma p-tau217 consistently aligns with cognitive status in A+ individuals and better reflects CSF biomarker abnormalities, reducing discrepancies between clinical and biochemical findings. Its association with baseline and future memory decline highlights its diagnostic and prognostic value, particularly when CSF analysis or PET is unavailable.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"53 1","pages":"e213676"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065881","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Factors Associated With Relapse in Patients With Neurosarcoidosis.","authors":"Louisa Nitsch,Dirk Skowasch,Edouard Courtin,Pierre Duffau,Carolin Otto,Klemens Ruprecht,Christine Dambietz,Michael Heming,Gerd Meyer Zu Hörste,Keld-Erik Byg,Christine Kindler,Julia Gutzwiller,Roxanne Pretzsch,Anne-Katrin Proebstel,Johannes M Weller,Julian Zimmermann","doi":"10.1212/wnl.0000000000213705","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213705","url":null,"abstract":"BACKGROUND AND OBJECTIVESFive to 10 percent of patients with sarcoidosis show involvement of the nervous system. Given the serious nature of neurosarcoidosis and the high risk of irreversible neurologic damage, rapid diagnosis, accurate prognostication, and individualized treatment are crucial. The aim of this study was to identify factors associated with relapses in neurosarcoidosis.METHODSIn this multicenter retrospective cohort study, patients diagnosed with possible, probable, or definite neurosarcoidosis between January 1, 2010, and June 30, 2024, at 6 tertiary neuroimmunology centers across Europe were included. Patients were identified from the respective hospital-based databases by the participating centers. Clinical presentation, imaging results, CSF analysis, and immunosuppressive therapies were evaluated. Predictors of relapse, defined as clinical relapse or progression, or new MRI lesions on follow-up ≥2 months after initial manifestation, were analyzed with log-rank tests and Cox regression models, and therapeutic strategies in clinical practice were assessed. The association between identified risk factors (RFs) and therapeutic strategies was explored.RESULTSA total of 174 patients with neurosarcoidosis were included with a median follow-up of 24 months (interquartile range 12-48.8). The mean age was 48.1 years, and 57.5% were female. CNS parenchymal lesions including encephalitis, myelitis, and optic neuritis (hazard ratio [HR] 2.3, 95% CI 1.3-4.2); CSF-specific oligoclonal bands (OCBs) (HR 2.1, 95% CI 1.3-3.6); CSF glucose <40 mg/dL (HR 2, 95% CI 1.1-3.4); and CSF protein ≥1,000 mg/dL (HR 2.1, 95% CI 1.3-3.5) were identified as RFs of relapse. Patients with 3-4 RFs had a median relapse-free survival of 12 months (95% CI 4.3-19.7), compared with 36 months (95% CI 24.3-47.7) in patients with 1-2 RFs and 120 months (95% CI 2.1-237.9) in patients without RFs (p < 0.01). The likelihood of treatment escalation increased with the number of RFs, from 14.3% in patients without RFs to 28.2% and 50% in patients with 1-2 or 3-4 RFs, respectively (p < 0.01).DISCUSSIONThe identification of specific RFs, including parenchymal lesions, OCBs, CSF glucose <40 mg/dL, and CSF protein ≥1,000 mg/dL, enables better prognostication and might inform individualized treatment approaches. Patients with multiple RFs are at greater risk of relapse, possibly suggesting the need for more aggressive therapies.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"14 1","pages":"e213705"},"PeriodicalIF":9.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From Transient Attack to Persistent Fatigue: Cause or Consequence?","authors":"Nina A Hilkens,Ewoud J Van Dijk","doi":"10.1212/wnl.0000000000213714","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213714","url":null,"abstract":"","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"121 1","pages":"e213714"},"PeriodicalIF":9.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143982531","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Fatigue Following Transient Ischemic Attack: A Prospective Cohort Study.","authors":"Birgitte Hede Ebbesen,Simon Grøntved,Jakob Nebeling Hedegaard,Søren P Johnsen,Jane Andreasen,Krystian Figlewski,Mirko Porobic,Michael Skovdal Rathleff,Boris Modrau","doi":"10.1212/wnl.0000000000213605","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213605","url":null,"abstract":"BACKGROUND AND OBJECTIVESBy definition, patients with transient ischemic attack (TIA) should not have residual symptoms beyond 24 hours. However, preliminary evidence indicates lasting challenges such as fatigue. It is unknown who develops fatigue, and the extent. This knowledge is required to develop evidence-based support for patients. We aimed to explore fatigue up to 12 months after TIA and determine what characterizes patients who experience pathologic fatigue.METHODSThis is a prospective cohort study including patients with TIA diagnosed at a specialized stroke unit. Fatigue was measured using Multidimensional Fatigue Inventory (MFI-20) and Fatigue Severity Scale at 14 days (baseline) and 3, 6, and 12 months after discharge. The association between candidate prognostic factors and fatigue at 12 months was tested using linear regression models. We compared model performances using likelihood ratio (LR).RESULTSWe included 354 patients, of which 287 provided baseline responses (mean age 70.0 ± 11.1, 42.5% female). At baseline and 3, 6, and 12 months, the mean level of general fatigue on MFI was 12.3 ± 4.6, 11.9 ± 4.6, 11.4 ± 4.5, and 11.1 ± 4.5 and the proportion with pathologic fatigue (≥12 on the MFI-20 General Fatigue) was 61.3%, 53.5%, 54.0%, and 53.8%, respectively. The prevalence of acute infarction was evenly distributed between patients who reported fatigue and those who did not. Previous anxiety/depression was twice as common in the group that reported fatigue. The model including baseline level of fatigue, sex, age, and acute infarction was able to explain variability in the reported data to a statistical significantly higher extent, compared with the model only including sex, age, and acute infarction (p < 0.001, LR = 387.30).DISCUSSIONPathologic fatigue is common up to 12 months after TIA diagnosis. If patients report fatigue within 14 days after discharge, it is likely that this remains until 12 months. We found no indication of an association between the presence of acute ischemic lesions and fatigue. The prevalence of previous anxiety/depression was higher in the group that reported fatigue.TRIAL REGISTRATION INFORMATIONThe study is part of the Life After Stroke Cohort (ClinicalTrials.gov: NCT05234528). Registered February 1, 2022. First patient included February 2, 2022.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"11 1","pages":"e213605"},"PeriodicalIF":9.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143982513","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus: Report of the AAN Guidelines Subcommittee.","authors":"Maryam Oskoui,Tracie Anne Caller,Julie A Parsons,Laurent Servais,Russell J Butterfield,Jyoti Bharadwaj,Sean C Rose,Benjamin Tolchin,Katie Puskala Hamel,Heather M Silsbee,James J Dowling","doi":"10.1212/wnl.0000000000213604","DOIUrl":"https://doi.org/10.1212/wnl.0000000000213604","url":null,"abstract":"This Evidence in Focus reviews the current evidence on the efficacy and adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) and presents clinical considerations regarding use. The author panel systematically reviewed available clinical trial data on delandistrogene moxeparvovec in patients with DMD. The risk of bias was evaluated using the American Academy of Neurology's 2017 therapeutic classification of evidence scheme. Safety information, regulatory decisions, and clinical context were also reviewed. Six clinical trials were identified, of which 4 had peer-reviewed data available. From the 4 studies with available data (2 Class I and 2 Class III), exposure data are available on 134 boys, of which 128 are ambulatory and aged ≥4 to <8 years. Both Class I studies failed to meet the primary functional motor outcome as assessed by change in the North Star Ambulatory Assessment score. Several secondary functional motor outcomes demonstrated improvement in the treatment group with small effect sizes, not meeting statistical significance from hierarchical analysis. Corticosteroid dose exposure was higher in the treatment group in the first 12 weeks after infusion, potentially contributing to measured differences between groups. Safety outcomes were similar across studies with multiple treatment-related adverse events, including peri-infusion effects, immune myositis and myocarditis, thrombocytopenia, and liver toxicity. One death has been reported in an individual who was treated with delandistrogene moxeparvovec outside of a trial. Despite not demonstrating efficacy in its primary outcome, delandistrogene moxeparvovec has been approved by the US Food and Drug Administration (FDA) for use in boys with DMD. This decision was supported by the relative safety of the product and secondary outcome measures data in the phase 3 clinical trial. As the drug may now be actively prescribed in the United States and other countries after FDA approval, providers should be aware of the limitations of the treatment and the need to monitor for immune-related side effects including myocarditis, liver injury, and thrombocytopenia, which may require expanded clinical infrastructure. Additional clinical trials and careful collection of real-world evidence from treated patients will be essential to establish short-term and long-term effectiveness and inform understanding of benefits and risks of delandistrogene moxeparvovec across the lifespan.","PeriodicalId":19256,"journal":{"name":"Neurology","volume":"14 1","pages":"e213604"},"PeriodicalIF":9.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143982452","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}