Neurology and Therapy最新文献

{"title":"Characteristics and Hospitalization Details of Patients with Advanced Parkinson's Disease Receiving Continuous Subcutaneous Infusion of Foslevodopa/Foscarbidopa: Real-World First-Year Descriptive Study from Japan.","authors":"Toru Baba, Toshiki Nozaki, Shunji Toya, Hideyuki Migita, Kairi Ri, Atsushi Takeda","doi":"10.1007/s40120-026-01016-9","DOIUrl":"https://doi.org/10.1007/s40120-026-01016-9","url":null,"abstract":"<p><strong>Introduction: </strong>The real-world characteristics of patients with advanced Parkinson's disease (PD) who receive foslevodopa/foscarbidopa (LDp/CDp) continuous subcutaneous infusion therapy (CSCI) remain poorly understood. This study aimed to describe the characteristics and hospitalization details of patients with advanced PD hospitalized for LDp/CDp CSCI in Japan.</p><p><strong>Methods: </strong>This study was a retrospective descriptive analysis conducted using a hospital-based database provided by Medical Data Vision, Co. Ltd. Patients with advanced PD who underwent LDp/CDp CSCI during hospitalization between July 2023 and June 2024 were enrolled. The index date was defined as the date of the first LDp/CDp prescription. Baseline patient characteristics, levodopa-equivalent dose (LED), number of pills/transdermal patches, medication classes for concomitant PD medications, and non-motor symptom medications assessed within 4 weeks prior to and including the index date, along with hospitalization details, were collected.</p><p><strong>Results: </strong>We enrolled 96 patients with a mean age of 65.6 years [standard deviation (SD), 10.2], of whom 58 (60.4%) were female. The median Barthel Index was 90.0 [interquartile range (IQR): 44.2], and the hospitalization duration was 15.0 days (IQR: 9.0). Eighteen patients (18.8%) discontinued LDp/CDp CSCI during hospitalization. The mean LED of any PD medications prior to LDp/CDp CSCI initiation, the mean number of PD medication classes, and the daily pill/transdermal patch count for PD medications were 1,111.0 mg/day (SD 575.7), 3.9 (SD 1.3), and 10.6 (SD 5.0), respectively. Except for levodopa (L-Dopa), dopamine agonists (78.1%) were the most commonly used concomitant PD medications.</p><p><strong>Conclusions: </strong>In real-world clinical practice in Japan, patients with LDp/CDp CSCI tend to be younger and have preserved activities of daily living. Our results implied that patients are often managed with multiple concomitant PD medications rather than with L-Dopa dose titration prior to initiation. Further research is warranted to maximize the benefits of LDp/CDp CSCI and assess its real-world impact on pill burden.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148892279","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":"Smartphone-Based Multimodal Digital Biomarker Integration for Parkinson's Disease Screening and Diagnostic Support.","authors":"Kyungsung Lee, Han-Joon Kim, Jung Hwan Shin, Seungmin Lee, Su Hyeon Ha, Chanhee Jeong, Kyung Ah Woo, Dasom Lee, Myungjun Lee, Joonsang Jo, ZunHyan Rieu, Yoohun Noh","doi":"10.1007/s40120-026-01023-w","DOIUrl":"https://doi.org/10.1007/s40120-026-01023-w","url":null,"abstract":"<p><strong>Introduction: </strong>Timely identification of Parkinson's disease (PD) is often delayed because of clinical heterogeneity and limited awareness of early symptoms. Digital biomarkers obtained via smartphones offer scalable screening potential. However, unimodal assessments may lack sufficient sensitivity or specificity given the multidimensional nature of PD. The aim of this study was to develop and validate a smartphone-based, multimodal digital biomarker framework for PD screening and diagnostic support.</p><p><strong>Methods: </strong>The study progressed through two phases: an initial version [n = 368; 233 PD, 135 healthy controls (HC)] was used for data-driven task refinement, and a final version (n = 296; 204 PD, 92 HC) containing optimized motor (Touch, Swipe, Balloon, Spiral, Wave), visual, and speech tasks and a refined questionnaire task was evaluated. Feature selection and speech subtask selection were performed exclusively within the training set using stratified cross-validation. Random Forest and XGBoost classifiers were trained using (1) single-task features, (2) all-task multimodal features, and (3) selected task subsets. The primary outcome was area under the receiver operating characteristic curve (AUROC) on the independent test set.</p><p><strong>Results: </strong>In the final version, single-task models demonstrated heterogeneous performance (Random Forest AUROC range 0.5689-0.8397), with the questionnaire (0.8397) and Touch task (0.7789) performing best individually. The all-task multimodal model achieved AUROC 0.8620. A reduced multimodal subset combining Touch, Spiral, and questionnaire features yielded the highest discriminative performance (AUROC 0.9053). Additional feature- and task-level analyses showed significant multivariate group differences (Hotelling's T² p < 0.001) and stronger inter-feature association in PD compared with HC, providing interpretability.</p><p><strong>Conclusion: </strong>A smartphone-only multimodal digital biomarker framework achieved high discrimination between PD and controls. Multimodal integration outperformed unimodal approaches, supporting the potential utility of scalable, smartphone-based tools for PD screening and diagnostic support. External validation in broader populations is warranted.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148875029","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":"Narrative Review: Efficacy, Safety, and Tolerability of Newer Third-Generation Antiseizure Medications for Focal Epilepsy: A New Benchmark in Treatment Outcomes?","authors":"William E Rosenfeld, Pavel Klein, Louis Ferrari, Vicente Villanueva","doi":"10.1007/s40120-026-01013-y","DOIUrl":"https://doi.org/10.1007/s40120-026-01013-y","url":null,"abstract":"<p><strong>Introduction: </strong>Focal seizures are the most common form of epilepsy. This narrative review analyzes the efficacy, safety, and tolerability of five newer third-generation antiseizure medications (ASMs) approved for treatment of focal seizures: lacosamide, perampanel, eslicarbazepine acetate, brivaracetam, and cenobamate.</p><p><strong>Methods: </strong>A search of PubMed and ClinicalTrials.gov identified phase 2/3 randomized controlled trials and long-term open-label extension (OLE) studies for the five most recently approved ASMs for focal seizures. Efficacy data were summarized, including median percent reduction in seizure frequency, ≥ 50% responder rates, ≥ 75% responder rates (when reported), and seizure freedom rates. Safety and tolerability were assessed based on treatment-emergent adverse events (TEAEs), serious adverse events, and discontinuation rates. Long-term efficacy, safety, and treatment retention rates were also summarized.</p><p><strong>Results: </strong>All five ASMs had statistically significant efficacy vs. placebo in short-term trials, with cenobamate demonstrating higher median percent seizure reduction (35.5-100%), ≥ 50% responder rates (40-81.6%), and seizure freedom rates (up to 52.4%) compared to the other four third-generation ASMs. In long-term OLEs, cenobamate had ≥ 50% responder rates > 70% and the highest estimated treatment retention rates at 3 years (61-65%) compared to lacosamide (51-53%), perampanel (46%), and brivaracetam (48%). Common TEAEs across all ASMs included dizziness, somnolence, fatigue, and headache. Brivaracetam and lacosamide had favorable profiles in multiple network meta-analyses. Specific safety considerations include psychiatric adverse events for perampanel and brivaracetam, and a risk of drug reaction with eosinophilia and systemic symptoms (DRESS) for cenobamate (significant risk reduction achieved using a gradual titration schedule).</p><p><strong>Conclusion: </strong>All third-generation ASMs were effective adjunctive therapies. Cenobamate was associated with higher responder and seizure freedom rates and higher long-term retention rates. Brivaracetam and lacosamide had favorable tolerability profiles. The notable seizure freedom rates with cenobamate suggest a potential new benchmark in focal seizure treatment. Comparator trials are needed to confirm possible efficacy and tolerability differences among third-generation ASMs.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148865084","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":"Artificial Intelligence in Neuromuscular Diseases: Opportunities for a Data-Scarce Field.","authors":"Shang Ma, Sushan Luo, Huahua Zhong","doi":"10.1007/s40120-026-01017-8","DOIUrl":"https://doi.org/10.1007/s40120-026-01017-8","url":null,"abstract":"<p><p>Neuromuscular diseases (NMDs) encompass over 800 distinct entities affecting approximately one in 1000 individuals worldwide, with progressive muscle weakness, atrophy, and motor impairment as primary clinical manifestations. The rarity of most NMDs creates fundamental challenges for artificial intelligence (AI) and machine learning (ML) applications that typically require large-scale datasets. In this narrative review we synthesize the literature published between 2018 and 2025 on AI applications across the NMD spectrum, organized by clinical application domain. We examine how AI has advanced diagnostic capabilities through genetic variant interpretation, muscle magnetic resonance imaging analysis, electromyography-based classification, and computational pathology. In disease monitoring and prognosis, wearable-derived digital biomarkers have achieved regulatory qualification (US Food and Drug Administration [FDA] and European Medicines Agency [EMA]) as clinical trial endpoints for Duchenne muscular dystrophy, while AI-driven survival models for amyotrophic lateral sclerosis (ALS) have been validated across 14 European centers. Proteomic and multi-omics analyses using ML have identified diagnostic panels for ALS. However, most reported models were developed and internally validated on single-center datasets, and few have undergone external or prospective validation or clinical implementation. Despite these achievements, research intensity varies dramatically across NMD subtypes, with ALS and Duchenne muscular dystrophy dominating while myotonic dystrophy, congenital myopathies, and metabolic myopathies remain virtually unexplored. Critical gaps persist in computational pathology, multi-center validation, and clinical translation. In this review, we discuss how federated learning, international collaborative networks (TREAT-NMD, Solve-RD, EURO-NMD), and foundation models can address these challenges, and propose directions for future AI-enhanced clinical studies in this data-scarce field.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148864867","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":"The Patient Experience of Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease Post-Diagnosis: Results from an International Survey.","authors":"Jonathan D Santoro, Gabrielle deFiebre, Julia Lefelar, Panayotes Demakakos, Danielle Hartigh, Magali Periquet, Khaleda Ahmadyar, Zara Morrison, Jacqueline Palace","doi":"10.1007/s40120-026-01010-1","DOIUrl":"https://doi.org/10.1007/s40120-026-01010-1","url":null,"abstract":"<p><strong>Introduction: </strong>Data on the post-diagnosis experience of individuals with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) and their caregivers are limited. We explored post-diagnosis treatment experience and satisfaction with care in individuals with MOGAD and associated caregiver burden.</p><p><strong>Methods: </strong>An online 57-question survey was distributed internationally to adults (aged ≥ 16 years) or caregivers assisting an adult or answering on behalf of a child (aged < 16 years) from October to December 2024.</p><p><strong>Results: </strong>Overall, 261 responses were collected (adults, 219; children, 42) across 27 countries. Of those prescribed a preventative treatment, 43% of adults (n = 71/167) and 42% of children (n = 13/31) stopped treatment; common reasons were side effects and lack of efficacy. Most adults (73%) and children (64%) reported difficulties accessing/trying to access MOGAD treatment and care. Substantial dissatisfaction was reported for chronic symptom management for adults (35%) and psychological/emotional support for children (31%). Compared with individuals with MOGAD (N = 261), caregivers (of adults, n = 61; of children, n = 42) were significantly more likely to report mental health impacts (26% vs 92%, respectively; p < 0.001).</p><p><strong>Conclusion: </strong>Persistent unmet care and support needs exist for individuals with MOGAD. Treatments were often stopped/changed, highlighting the need for more efficacious, tolerable, and approved therapies. Caregivers experience significant mental health impacts; improved support is needed.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148865164","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":"Wilson's Disease Coexisting with Narcolepsy Type 1: Evidence for a Multifactorial Cause of Excessive Daytime Sleepiness from a Case Report.","authors":"Zhenjing Xu, Yanxin Wang, Chunsheng Xu, Zhifeng Hou, Wenming Yang","doi":"10.1007/s40120-026-01011-0","DOIUrl":"https://doi.org/10.1007/s40120-026-01011-0","url":null,"abstract":"<p><strong>Background: </strong>Wilson's disease (WD) is an autosomal recessive disorder of copper metabolism that frequently involves the central nervous system and may be associated with secondary sleep disturbances. Excessive daytime sleepiness (EDS) in WD is typically attributed to metabolic or structural brain injury rather than primary sleep-wake dysregulation. Narcolepsy type 1 (NT1), caused by autoimmune loss of hypothalamic hypocretin neurons, is a distinct primary sleep disorder. The coexistence of WD and NT1 has not previously been reported.</p><p><strong>Case presentation: </strong>We describe a 23-year-old woman with a 15-year history of WD who developed long-standing EDS accompanied by emotionally triggered cataplexy, sleep paralysis, and hypnagogic hallucinations. Genetic analysis identified pathogenic ATP7B variants (p.R778L and p.R919G). Brain MRI revealed symmetric signal abnormalities involving the basal ganglia, midbrain nuclei, cerebellar dentate nuclei, and hypothalamus, suggesting chronic neurotoxicity. Comprehensive sleep evaluation supported a diagnosis clinically consistent with NT1, further corroborated by HLA-DQB1*06:02 positivity. Importantly, hepatic encephalopathy and other secondary causes of hypersomnolence were excluded.</p><p><strong>Conclusion: </strong>This case suggests the possibility that NT1 can coexist with WD as an independent primary sleep disorder rather than a secondary manifestation of metabolic or structural brain disease. The findings support a multifactorial neurobiological mechanism in which copper-related neurotoxicity and genetic susceptibility converge on hypothalamic injury. Clinicians should maintain a high index of suspicion for primary sleep-wake disorders when patients with WD present with cataplexy or other REM-related symptoms, as accurate differentiation has important implications for diagnosis, management, and prognosis. Infographic available for this article. INFOGRAPHIC.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148851397","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":"Safety and Effectiveness of Inebilizumab in Patients with Neuromyelitis Optica Spectrum Disorder: Interim Analysis of a Post-marketing Surveillance in Japan.","authors":"Kazuo Fujihara, Shinya Hirota, Muneyoshi Kudo, Hideaki Hida, Satoshi Yuki, Takashi Narisawa, Kyoko Kato","doi":"10.1007/s40120-026-01008-9","DOIUrl":"https://doi.org/10.1007/s40120-026-01008-9","url":null,"abstract":"<p><strong>Introduction: </strong>Inebilizumab is used in Japan for relapse prevention in aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). Nationwide post-marketing surveillance (PMS) is underway to evaluate the real-world safety and effectiveness of inebilizumab in Japanese patients with NMOSD.</p><p><strong>Methods: </strong>This PMS interim analysis assessed safety and effectiveness of inebilizumab in Japanese patients with NMOSD initiating treatment between June 2021 and November 2023, using data through June 2025.</p><p><strong>Results: </strong>The safety analysis set included 228 patients, and the effectiveness analysis set included 224 patients after excluding three patients from N-MOmentum (NCT02200770) and one without confirmed anti-AQP4 antibody positivity. Twenty-one (safety analysis set) and 20 (effectiveness analysis set) patients discontinued treatment. In the safety analysis set, 194 patients (85.1%) were female, and the mean ± standard deviation (SD) age was 54.2 ± 13.1 years. Adverse drug reactions (ADRs) occurred in 74 patients (32.5%); 31 (13.6%) were serious. Common ADRs were infections (14.5%, n = 33); serious infections (5.7%, n = 13) included COVID-19 (1.3%, n = 3), COVID-19 pneumonia (0.9%, n = 2), and urinary tract infection (0.9%, n = 2). Two fatal ADRs (interstitial lung disease, sudden cardiac death) occurred, for which a causal relationship with inebilizumab could not be excluded. In the effectiveness analysis set, the annualized relapse rate (95% CI) decreased from 0.40 (0.33-0.47) per person-year during the 2 years before inebilizumab initiation to 0.10 (0.07-0.14) per person-year after inebilizumab initiation. From baseline to 1 year, the proportion of patients receiving neither concomitant oral glucocorticoids nor immunosuppressants increased from 5.4% to 33.0%, and the mean ± SD oral glucocorticoid dose decreased from 11.7 ± 7.9 mg/day to 4.3 ± 4.7 mg/day.</p><p><strong>Conclusion: </strong>In this interim analysis, the safety profile of inebilizumab was consistent with prior evidence and no new safety concerns were identified. Inebilizumab reduced relapse rates and decreased concomitant use of oral glucocorticoids and immunosuppressants in patients with NMOSD.</p><p><strong>Umin clinical trials registry: </strong>UMIN000044431.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148851409","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":"Patient Preferences Toward CGRP-Targeting Preventive Migraine Treatments in Japan: Discrete Choice Experiment.","authors":"Hisaka Igarashi, Toru Yamazaki, Keigo Hanada, Hitomi Onomura, Hiroyuki Hozawa, Tatsunori Murata","doi":"10.1007/s40120-026-01015-w","DOIUrl":"https://doi.org/10.1007/s40120-026-01015-w","url":null,"abstract":"<p><strong>Introduction: </strong>To date, no Japanese study on migraine has evaluated how treatment characteristics affect patient preferences between novel gepants and subcutaneous or novel intravenous calcitonin gene-related peptide (CGRP) monoclonal antibodies. A survey using a discrete choice experiment (DCE) was therefore conducted to identify treatment preferences for CGRP-targeting preventive therapies in this population.</p><p><strong>Methods: </strong>Preventive-treatment attributes and levels were identified via a literature review, pivotal clinical trials, and international regulatory documents. These levels included those of CGRP-targeting therapies under development in Japan. A five-patient focus group was conducted to confirm patient understanding of the attribute descriptions and identify other potential attributes. Results from the focus group interview and from clinician input informed a questionnaire, which was revised following a pilot survey (N = 39), then an online main survey was conducted in May-June 2025 with eligible patients. A DCE was used to quantify patient preferences, and a conditional logit model was used to calculate preference weights and 95% confidence intervals.</p><p><strong>Results: </strong>The questionnaire included six attributes: 50% reduction rate of migraine frequency, speed of effect, effect on impairment of daily activities, frequency of side effects, frequency of injection-site reactions, and administration method. The main survey included 324 patients (79% women; mean age 47 years) who experienced an average of eight migraine days per month. The survey revealed that effect on impairment of daily activities was the most important attribute, followed by administration method, and frequency of injection-site reactions. Regarding attribute levels, oral CGRP-targeting formulations were preferred over injectables within the specific attribute combinations used in this DCE.</p><p><strong>Conclusions: </strong>These findings suggest that, under the design conditions used in this DCE, participants with migraine in Japan favored non-invasive oral CGRP-targeting treatments that effectively reduce impairment of daily activities.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148831060","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":"Assessment of Transition Plan Implementation and Provider Engagement Among Adults Living with Spinal Muscular Atrophy (SMA): Findings from a Cross-Sectional Survey.","authors":"Lauren E Eisenman, Angie R Wise, Mary A Curry","doi":"10.1007/s40120-026-01012-z","DOIUrl":"https://doi.org/10.1007/s40120-026-01012-z","url":null,"abstract":"<p><strong>Introduction: </strong>Advances in disease-modifying therapies have improved outcomes and survival in spinal muscular atrophy (SMA), resulting in a growing population of adolescents and young adults who must transition from pediatric to adult healthcare systems. Despite longstanding professional guidance supporting structured transition planning, limited data describe how these processes are implemented and experienced in SMA in the United States (U.S.). This study evaluated patient-reported experiences of transition planning and engagement with adult healthcare providers among young adults living with SMA.</p><p><strong>Methods: </strong>A cross-sectional survey was distributed by email invitation to U.S. adults aged 18-29 years with 5q-SMA listed in the Cure SMA membership database, and interested individuals participated voluntarily. Descriptive analyses summarized transition status, exposure to structured transition elements, coordination practices, and experiences with adult providers. Respondents who had initiated transition were categorized by self-reported transition experience as Excellent/Very good/Good (EVG) or Fair/Poor (FP).</p><p><strong>Results: </strong>Of 156 responses received (30.4% response rate), 137 met inclusion criteria, and 122 had initiated or completed transition. Among these, 59.0% reported fully transitioning to adult care. Exposure to structured preparation elements was limited overall but more commonly reported by EVG respondents compared with FP respondents, including readiness assessments (28.2% vs. 6.8%), discussions about transition planning (51.3% vs. 17.8%), and receipt of written transition plans (17.5% vs. 7.7%). FP respondents more commonly reported self-coordinating their transition (36.4% vs. 23.1%) and major challenges engaging with adult providers (26.3% vs. 4.6%) compared with EVG respondents. Nearly all respondents (98.0%) reported difficulty accessing adult clinicians with SMA-specific expertise.</p><p><strong>Conclusions: </strong>Transition experience among young adults with SMA was more closely associated with cumulative exposure to structured preparation elements than with a single transfer event. Broader implementation of provider-led transition practices may improve patient experience, strengthen engagement in adult SMA care, and support care coordination within adult systems where disease-specific expertise remains limited.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148795947","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":"Correction: Utility of Multi‑Analyte Protein Assay to Distinguish Multiple Sclerosis Clinical Relapse from Pseudoexacerbation.","authors":"Darin T Okuda, Tatum M Moog, Morgan C McCreary, Isabella J Huddleston, Crystal M Wright, Katy W Burgess, Jose R Santoyo, Peter V Sguigna, Olaf Stüve, Diem H Tran","doi":"10.1007/s40120-026-01005-y","DOIUrl":"https://doi.org/10.1007/s40120-026-01005-y","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148762345","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}