Journal of Neurology最新文献

{"title":"Randomised feasibility study evaluating eye movement desensitisation and reprocessing therapy for functional neurological disorder (MODIFI).","authors":"Sarah R Cope, Jared G Smith, Sharif El-Leithy, Serena Vanzan, Patricia Hogwood, Dawn Golder, Kati Jane Turner, Maeve Crowley, Jo Billings, Susannah Pick, Caitlin Pentland, Mark J Edwards","doi":"10.1007/s00415-025-13219-5","DOIUrl":"10.1007/s00415-025-13219-5","url":null,"abstract":"<p><strong>Background: </strong>Functional neurological disorder (FND) is a common neurological presentation with symptoms such as seizures, walking difficulties, limb weakness and cognitive difficulties. Treatments for FND include physiotherapy and psychological therapy. Eye movement desensitisation and reprocessing therapy (EMDR) is a therapy designed to reduce disturbance associated with distressing or traumatic memories. Case report evidence suggests possible benefit for people with FND. This randomised feasibility study aimed to assess whether a large-scale trial evaluating EMDR for FND would be feasible and acceptable.</p><p><strong>Methods: </strong>Fifty participants with FND were randomised to either FND-focused EMDR plus standard neuropsychiatric care (NPC) or NPC alone. Feasibility criteria were recruitment rate, intervention adherence, and outcome measure completion. Assessment of safety was also examined, as well as therapy satisfaction. Participants completed questionnaires at baseline, 3 months, 6 months and 9 months. FND symptoms were assessed using Ecological Momentary Assessment at each time point.</p><p><strong>Results: </strong>Recruitment rate was 58%, intervention adherence was 88%, and outcome measure completion was 68% for Ecological Momentary Assessment and 76% for questionnaires at 9-month follow-up. Participants experienced functional motor symptoms (80%), functional seizures (64%), and cognitive symptoms (32%). Participants receiving EMDR + NPC reported greater satisfaction and greater FND improvement compared to NPC. Questionnaire data suggested greater reductions in PTSD, depression, anxiety, dissociation, disability and healthcare-use for EMDR + NPC.</p><p><strong>Discussion: </strong>The study demonstrated that an FND-specific protocol for EMDR was feasible and acceptable. Potential positive effects on FND symptoms, mental health, disability, and healthcare utilisation were found. A full-scale trial is warranted to establish efficacy.</p><p><strong>Trial registration: </strong>NCT05455450 ( www.</p><p><strong>Clinicaltrials: </strong>gov ).</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"493"},"PeriodicalIF":4.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12238153/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584199","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Therapeutic strategies targeting complement in myasthenia gravis patients.","authors":"Yinxiang Wang, Jianfei Nao, Yingjie Duan, Zijian Li, Juan Feng","doi":"10.1007/s00415-025-13225-7","DOIUrl":"10.1007/s00415-025-13225-7","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an acquired autoimmune disorder characterized by impaired neuromuscular junction transmission, leading to fluctuating muscle weakness. With the progressive identification of MG-related antibodies, such as acetylcholine receptor (AChR) antibodies and muscle-specific kinase (MuSK) antibodies, coupled with advancements in antibody detection technology, the use of these diagnostic markers has become widely accessible in clinical practice. This has facilitated the detection of key biomarkers and enabled the study of targeted therapeutic interventions aimed at addressing the underlying production of MG pathogenic antibodies and their various stages of pathogenicity. Among the emerging therapeutic strategies, complement-targeting drugs have garnered significant attention from medical researchers because of their ability to reduce complement activation and inhibit autoimmune-mediated tissue damage. Inhibition of the terminal complement cascade has demonstrated efficacy in reducing disease severity and improving clinical outcomes, particularly in refractory cases of MG. This review summarizes the underlying mechanisms of complement activation in MG, evaluates the current therapeutic landscape, including both approved and investigational complement inhibitors and discusses the safety considerations of their application.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"489"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222297/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540582","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative effectiveness of teriflunomide and ocrelizumab on smoldering activity in multiple sclerosis: an observational study in the Swiss Multiple Sclerosis Cohort.","authors":"Alessandro Cagol, Sabine Schaedelin, Mario Ocampo-Pineda, Pascal Benkert, Lester Melie-Garcia, Ludovico Luchetti, Özgür Yaldizli, Johanna Oechtering, Marcus D'Souza, Bettina Fischer-Barnicol, Stefanie Müller, Sebastian Finkener, Jochen Vehoff, Giulio Disanto, Andrew Chan, Caroline Pot, Chiara Zecca, Tobias Derfuss, Johanna M Lieb, Michael Diepers, Franca Wagner, Renaud Du Pasquier, Patrice H Lalive, Emanuele Pravatà, Olaf Chan-Hi Kim, Robert Hoepner, Patrick Roth, Claudio Gobbi, David Leppert, Marco Battaglini, Ludwig Kappos, Maria Pia Sormani, Jens Kuhle, Cristina Granziera","doi":"10.1007/s00415-025-13221-x","DOIUrl":"10.1007/s00415-025-13221-x","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to compare the effects of teriflunomide and ocrelizumab on clinical and MRI endpoints related to smoldering activity in relapsing-remitting multiple sclerosis (RRMS).</p><p><strong>Methods: </strong>In this observational, longitudinal, multicenter study, we included 128 people with RRMS (pwRRMS) treated with teriflunomide and 495 treated with ocrelizumab. Outcomes included time to progression independent of relapse activity (PIRA). In a subset, we also assessed brain volume loss (BVL), longitudinal changes in diffusion tensor imaging (DTI) metrics, and the burden of paramagnetic rim lesions (PRLs). Propensity score matching was used for between-group comparisons.</p><p><strong>Results: </strong>Over a median follow-up of 3.1 years in the ocrelizumab group and 1.9 years in the teriflunomide group, there were no significant differences in the risk of PIRA (HR for teriflunomide vs. ocrelizumab: 0.80 [95%-CI:0.40-1.60]; p = 0.53). PwRRMS treated with teriflunomide exhibited lower annualized rates of BVL (-0.80 [95%-CI: -0.91; -0.69] vs. -1.06 [95%-CI: -1.25; -0.86]; p = 0.025) and gray matter volume loss (-0.92 [95%-CI: -1.05; -0.79] vs. -1.20 [95%-CI: -1.43; -0.97]; p = 0.035). No differences were observed in DTI metrics or PRL count.</p><p><strong>Conclusions: </strong>This real-world study suggests that teriflunomide shows similar efficacy to ocrelizumab on smoldering activity, with a potentially greater effect in reducing BVL. Further research is needed to confirm these findings and understand their long-term implications.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"491"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222313/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144553788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Frailty diminishes functional outcome in patients with nonaneurysmal subarachnoid hemorrhage: a dual specialized neurovascular center analysis.","authors":"Larissa Penner, Anna-Laura Potthoff, Tim Lampmann, Rebecca Heinz, Johannes Lemcke, Motaz Hamed, Florian Gessler, Hartmut Vatter, Patrick Schuss, Alexis Hadjiathanasiou, Matthias Schneider","doi":"10.1007/s00415-025-13227-5","DOIUrl":"10.1007/s00415-025-13227-5","url":null,"abstract":"<p><strong>Objective: </strong>Frailty is increasingly recognized as a significant prognostic factor in various conditions. However, its impact on outcomes following spontaneous, nonaneurysmal subarachnoid hemorrhage (naSAH) remains unclear. This study aimed to assess the association between pre-existing frailty and functional outcomes in patients with naSAH.</p><p><strong>Methods: </strong>The study cohort was made up of 257 patients treated for naSAH at two neurovascular centers between 2012 and 2021. Frailty prior to naSAH was assessed using the modified frailty index (mFI), with patients classified as nonfrail (mFI 0-1) or frail (mFI ≥ 2). Functional outcomes at 6 months were evaluated using the modified Rankin Scale (mRS), categorized as favorable (mRS 0-2) or unfavorable (mRS 3-6). A multivariable logistic regression analysis was performed to identify independent predictors of unfavorable outcomes.</p><p><strong>Results: </strong>Among 257 naSAH patients, 56 (22%) were classified as frail (mFI ≥ 2) before ictus. At the 6-month follow-up, unfavorable outcomes were observed in 17 of the 56 frail patients (30%) compared to 21 of 201 nonfrail patients (10%) (p = 0.001). In addition to established negative prognostic factors such as delayed cerebral ischemia (p < 0.001) and poor-grade naSAH (Hunt & Hess grades III-IV; p = 0.001), multivariable analysis identified frailty (p = 0.03) as an independent and significant predictor of unfavorable functional outcomes.</p><p><strong>Conclusions: </strong>Frailty prior to hemorrhage, as determined by an mFI of ≥ 2, was associated with poor functional outcomes at 6 months in patients with naSAH. These findings underscore the importance of incorporating frailty assessments into early prognostic evaluations to guide patient management and counseling.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"488"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222263/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multiparametric MRI-based biomarkers in the non-fluent and semantic variants of primary progressive aphasia.","authors":"Marco Michelutti, Hans-Jürgen Huppertz, Heiko Volkmann, Sarah Anderl-Straub, Daniele Urso, Benedetta Tafuri, Salvatore Nigro, Paolo Manganotti, Leonie Werner, Jolina Lombardi, Markus Otto, Giancarlo Logroscino, Hans-Peter Müller, Jan Kassubek","doi":"10.1007/s00415-025-13215-9","DOIUrl":"10.1007/s00415-025-13215-9","url":null,"abstract":"<p><strong>Background: </strong>The non-fluent (nfPPA) and semantic (svPPA) variants of primary progressive aphasia exhibit distinct clinical features. We investigated whether diffusion tensor imaging (DTI) and atlas-based volumetry (ABV) could reveal divergent patterns of longitudinal changes in brain white matter microstructure and gray matter volumes.</p><p><strong>Methods: </strong>MRI datasets from 29 nfPPA, 27 svPPA, and 39 controls were analyzed. White matter fractional anisotropy (FA) differences were assessed using unbiased Whole Brain-based Spatial Statistics (WBSS) and Tract-Wise Fractional Anisotropy Statistics (TFAS). Gray matter volumetric differences were calculated by Atlas-Based Volumetry (ABV). A subset of 10 nfPPA and 6 svPPA patients underwent longitudinal MRI at 12 months. FA maps were correlated with disease severity (FTLD-CDR sum of boxes). A random forest classifier validated tracts of interest (TOI) and structures of interest (SOIs) selection as a proof-of-concept.</p><p><strong>Results: </strong>At baseline, nfPPA showed frontal, callosal, and temporal white matter degeneration, while the left inferior longitudinal fasciculus (ILF) was predominantly involved in svPPA. Longitudinally, nfPPA exhibited frontal, callosal, and posterior temporal progression, while svPPA showed localized antero-posterior ILF progression. ABV aligned with the DTI analyses, demonstrating volumetric reductions in the frontal lobe for nfPPA and in temporal lobe and subcortical limbic structures in svPPA. Sub-clusters of white matter damage progression correlated with worsening FTLD-CDR scores. Random forest analysis identified the most discriminative TOIs and SOIs.</p><p><strong>Conclusions: </strong>Distinct degeneration patterns emerged across nfPPA and svPPA, supporting early differential diagnosis and correlating with disease worsening. These findings support the utility of combined DTI and ABV in tracking disease progression.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"490"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Insight into NeuroCOVID: neurofilament light chain (NfL) as a biomarker in post-COVID-19 patients with olfactory dysfunctions.","authors":"Fiorenza Pennacchia, Federica Zoccali, Carla Petrella, Giuseppina Talarico, Eqrem Rusi, Maria Antonella Zingaropoli, Wael Abu Ruqa, Giuseppe Bruno, Rosamaria Capuano, Alexandro Catini, Corrado Di Natale, Antonio Minni, Christian Barbato","doi":"10.1007/s00415-025-13222-w","DOIUrl":"10.1007/s00415-025-13222-w","url":null,"abstract":"<p><p>The term NeuroCOVID was coined to describe the neurological consequences observed in COVID-19 patients. Numerous patients infected with SARS-CoV-2 reported olfactory dysfunction as the first symptom preceding clinical manifestations, such as cough and fever, or even the only symptom, suggesting the sudden loss of smell or hyposmia as an important predictive factor for COVID-19 infection. Several patients developed long-term olfactory impairment, but to date there is not available a biochemical diagnosis of anosmia. The aim of this pilot study is to investigate the association between neurofilament light-chain (NfL) serum levels and the olfactory dysfunctions in post-COVID-19 patients. This study recruited patients who developed COVID-19 between January 2020 and August 2021. They were evaluated between October 2022 and March 2023 by Sniffin' Sticks tests to investigate deficits of odor identification, discrimination, and threshold and serum NfL biomarker measurement to assess a neuronal damage. Out of 27 patients, 11 were affected by post-viral permanent olfactory dysfunction (named Od-post-COVID-19) and 16 healed from the infection without residual Od problem, as a control group. We observed an increased levels of NfL 16.02 ± 1.91 pg/mL in Od-post-COVID-19, suggesting that NfL to be recognized as a biomarker of post-viral olfactory dysfunction, supporting the diagnostic process of NeuroCOVID, joined with other well-known neurological biomarkers and/or innovative investigative approaches.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 7","pages":"484"},"PeriodicalIF":4.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12213847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540579","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Genetic variation of low-to-medium-affinity Fc-gamma receptors in Guillain-Barré syndrome.","authors":"Sander J van Tilburg, Selin Koçer, Judy Geissler, Wouter van Rijs, Anne P Tio-Gillen, Michael W T Tanck, Willem-Jan R Fokkink, Pieter A van Doorn, Bart C Jacobs, Sietse Q Nagelkerke, Ruth Huizinga","doi":"10.1007/s00415-025-13216-8","DOIUrl":"10.1007/s00415-025-13216-8","url":null,"abstract":"<p><strong>Introduction: </strong>Fc-gamma receptors (FcγRs) are important for the effector functions of immunoglobulin G (IgG) and are therefore expected to play a role in the pathophysiology of Guillain-Barré syndrome (GBS). The FCGR2/3 locus, which encodes low-to-medium-affinity FcγRs, contains extensive genetic variation. We hypothesized that genetic variation in the FCGR2/3 locus influences GBS susceptibility, muscle weakness, outcomes, and the pharmacokinetics of intravenous immunoglobulin (IVIg).</p><p><strong>Methods: </strong>Copy number variation and single nucleotide polymorphisms in the FCGR2/3 locus were studied using multiplex ligation-dependent probe amplification (MLPA). The study cohort consisted of 467 GBS patients and 919 healthy controls of European descent. Severe weakness was defined as an MRC sum score < 40 at nadir. The increase in serum IgG one or two weeks after start of IVIg treatment was determined.</p><p><strong>Results: </strong>No significant associations were found between genetic variation in the FCGR2/3 locus and susceptibility to GBS. However, in patients with an antecedent Campylobacter jejuni infection, a higher frequency of three or more FCGR3A copies was observed compared to healthy controls (p = 0.023). FCGR3A copy numbers were also associated with more severe disease (OR = 2.02; 95% CI = 1.00-4.12), even after correcting for age and positive C. jejuni serology. No association was found between FCGR2/3 variants and the ability to walk unaided in time-to-event analyses. In addition, the pharmacokinetics of IVIg were not affected by genetic variation in the FCGR2/3 locus.</p><p><strong>Conclusion: </strong>Overall, FCGR2/3 polymorphisms are not associated with susceptibility to GBS or response to IVIg treatment. However, associations may exist in specific subgroups, as demonstrated in patients with a preceding C. jejuni infection who more frequently carry a duplication in FCGR3A.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 7","pages":"487"},"PeriodicalIF":4.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12213970/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540578","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clonidine for the management of crises in stiff-person syndrome.","authors":"Salvatore Maria Lima, Nicasio Rini, Andrea Calì, Filippo Brighina, Vincenzo Di Stefano","doi":"10.1007/s00415-025-13226-6","DOIUrl":"10.1007/s00415-025-13226-6","url":null,"abstract":"<p><p>Stiff-person syndrome (SPS) is a rare autoimmune neurological disorder characterized by high titers of antibodies against glutamic acid decarboxylase (GAD) and impaired GABAergic inhibitory neurotransmission. If not promptly treated, SPS progresses to disability with high risk of respiratory insufficiency; therefore, it is essential to apply the best therapeutic regimens since the beginning, both in the chronic and in the emergency setting during exacerbations. To date, there are no defined guidelines for the treatment of this rare disease. In this study, we report the case of a woman affected by refractory SPS who benefited from clonidine with immediate resolution of stiffness and SPS crisis.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 7","pages":"485"},"PeriodicalIF":4.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative efficacy and safety of irreversible (rasagiline) and reversible (safinamide) monoamine oxidase inhibitors as add-on therapy for Parkinson's disease.","authors":"Marwah Bintay Khalid, Faizan Shahzad, Momina Riaz Siddiqui, Muhammad Zain Ul Abedin, Saad Hulou, Besher Shami, Syed Ijlal Ahmed","doi":"10.1007/s00415-025-13230-w","DOIUrl":"10.1007/s00415-025-13230-w","url":null,"abstract":"<p><strong>Background: </strong>Parkinson's disease (PD) is a progressive, neurodegenerative condition caused by progressive loss of dopaminergic neurons of the substantia nigra. Safinamide and rasagiline are both novel medications that are indicated for PD. Safinamide (reversible) and rasagiline (Irreversible) are selective monoamine oxidase B inhibitors which work by decreasing the degradation of dopamine in the brain.</p><p><strong>Objective: </strong>This network meta-analysis aimed to examine the efficacy and safety of both drugs as add-on therapy in patients with Parkinson's disease.</p><p><strong>Methodology: </strong>A systematic literature search of PubMed, Embase, and Cochrane databases was conducted in September 2024. Primary outcomes were Changes in the Unified Parkinson's Disease Rating Scale (UPDRS-III) and adverse effects. Standardized mean difference (SMD) and odds ratio (OR) were calculated with 95% confidence intervals. Surface Under Cumulative Ranking Curve (SUCRA) was used to compare individual interventions. The Cochrane risk-of-bias tool for randomized trials (RoB 2) was used to assess the risk of bias in studies.</p><p><strong>Results: </strong>The search query resulted in 557 studies. After screening, 15 studies were included in the final analysis, totaling 5676 participants. Safinamide (100 mg) was associated with the highest change in UPDRS-III scores (SMD = 0.3007, 95% CI = [0.1710-0.4304], z-score = 4.54, p value =  < 0.0001, I2 = 55.8%, SUCRA = 71.17%), with rasagiline (1 mg) being a close contender with a SUCRA of 70.64%. Safinamide (50 mg) had the lowest odds of serious adverse events (SAEs) with OR = 0.4394 (95% CI = [0.2231-0.8652], z-score = - 2.38, p value = 0.0174, I2 = 0%, SUCRA = 99.34%). Safinamide (100 mg) had the second-lowest odds of SAEs (OR = 0.9575, 95% CI = [0.6520-1.4061], z-score = - 0.22, p value = 0.8246, I2 = 0%, SUCRA = 66.96%). Almost all the studies had a low risk of bias.</p><p><strong>Conclusion: </strong>Safinamide (100 mg) has good efficacy outcomes, whereas safinamide (50 mg) has favorable safety outcomes as compared to rasagiline for add-on therapy for PD.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 7","pages":"486"},"PeriodicalIF":4.8,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Equity in neuromuscular research: a 20-year analysis of race, ethnicity, sex, and age representation.","authors":"Lorenzo Fontanelli, Gabriele Vadi, Gabriele Bellini, Andrea Cossu, Gabriele Siciliano","doi":"10.1007/s00415-025-13208-8","DOIUrl":"10.1007/s00415-025-13208-8","url":null,"abstract":"<p><strong>Objectives: </strong>We conducted a systematic analysis of studies on neuromuscular diseases registered on ClinicalTrials.gov over the last 20 years to assess disparities in study populations.</p><p><strong>Methods: </strong>Data from interventional and observational neuromuscular disease studies initiated between January 1, 2004, and December 31, 2024, were retrieved from ClinicalTrials.gov and PubMed/MEDLINE. Collected variables included participant race, ethnicity, sex, eligible age range, mean and median ages, as well as study funding source, start year, and phase. These variables were analyzed to evaluate disparities in race, ethnicity, and age across studies and over time.</p><p><strong>Results: </strong>A total of 2166 studies were screened, with 462 meeting inclusion criteria, encompassing data from 37,131 participants. Most participants were male (61.4%), White (83.5%), and non-Hispanic/Latino (87.6%). While the proportion of studies reporting race and ethnicity increased over time (p < 0.001 and p = 0.001, respectively), the racial and ethnic composition of participants remained unchanged (p = 1). Studies on X-linked recessive disorders (i.e., Duchenne muscular dystrophy (DMD), Becker muscular dystrophy, and spinal and bulbar muscular atrophy (SMA)) predominantly excluded female participants. Regarding age accessibility, 37.9% of studies allowed children. Similarly, trial accessibility for older adults was limited. Even in studies with broader age eligibility, mean and median participant ages clustered around midlife, with underrepresentation at both age extremes. Notably, about half of DMD and SMA studies excluded participants over 16 and 18 years, respectively.</p><p><strong>Conclusion: </strong>Significant disparities persist in race, ethnicity, and age representation in neuromuscular disease clinical research, highlighting the need for more inclusive study designs.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 7","pages":"483"},"PeriodicalIF":4.8,"publicationDate":"2025-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144528422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}