Neurology and Therapy最新文献

{"title":"Effectiveness and Safety of Low-Sodium Oxybate in Participants with Narcolepsy: Primary Results from the DUET Study.","authors":"Logan D Schneider, Chad M Ruoff, David T Plante, Deborah A Nichols, Teresa L Steininger, Douglas S Fuller, M Todd Kirby, Sarah Akerman, Jessica K Alexander, Marisa Whalen, Alyssa Cairns","doi":"10.1007/s40120-026-00921-3","DOIUrl":"10.1007/s40120-026-00921-3","url":null,"abstract":"<p><strong>Introduction: </strong>A phase 4, prospective, open-label study of low-sodium oxybate (LXB) in narcolepsy (type 1 [NT1] or 2 [NT2]) or idiopathic hypersomnia included novel symptom outcomes important to patients (Jazz DUET; NCT05875974; registered 16 May 2023). Primary results from the narcolepsy cohort, including LXB effectiveness (nighttime sleep/daytime symptoms/overall disease severity) and safety are reported here.</p><p><strong>Methods: </strong>DUET included screening, 8-day baseline (BL; off-LXB), 2-8-week LXB dose titration/optimization, 2-week stable-dose, 8-day end-of-treatment (EOT; on-LXB), and safety follow-up periods. At BL and EOT, participants underwent nocturnal polysomnography (PSG) and completed Epworth Sleepiness Scale (ESS; primary endpoint), Narcolepsy Severity Scale (NSS [NT1]; NSS-2 [NT2]), and Patient Global Impression of Severity (PGI-S) and Change (PGI-C); eDiaries for sleep quality and cataplexy (NT1 only) were completed daily for 8 days before PSGs. Least-squares mean (LSM) changes were adjusted for BL values.</p><p><strong>Results: </strong>Thirty-four participants completed the study and were analyzed (NT1, n = 16; NT2, n = 18); LSM (SE) change in ESS score (BL to EOT), - 7.7 (0.9), P < 0.0001. At EOT versus BL, transitions to lighter stages of sleep decreased (LSM [SE] - 13.1 [2.9], P < 0.0001), N3 duration increased (45.0 [8.8] min, P < 0.0001), and nocturnal awakenings decreased (- 3.2 [0.9], P = 0.0013). LSM [SE] changes in NSS and NSS-2 scores were - 19.7 (2.7) and - 11.3 (1.6). Most participants reported improved sleep quality and overall narcolepsy disease and fewer cataplexy attacks. Treatment-emergent adverse events were consistent with the known LXB safety profile.</p><p><strong>Conclusion: </strong>DUET study results demonstrated novel nighttime sleep/daytime symptom improvements in participants with narcolepsy treated with open-label LXB.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier, NCT05875974.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1613-1641"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396106/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147777305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utilization of Non-pharmacological Interventions in Rett Syndrome: A Systematic Review of the Literature on Supportive Care Management.","authors":"Nazia Rashid, Safiuddin Shoeb Syed, Krithika Rajagopalan, Vinod Kumar Yakkala, Mirko Sikirica, Ismaeel Yunusa","doi":"10.1007/s40120-026-00962-8","DOIUrl":"10.1007/s40120-026-00962-8","url":null,"abstract":"<p><strong>Introduction: </strong>Rett syndrome (RTT) is a rare neurodevelopmental disorder characterized by early childhood loss of mobility, reduced verbal communication, and behavioral impairment. Although non-pharmacological supportive interventions are widely used, there is a need to better understand their role in RTT care globally.</p><p><strong>Methods: </strong>Following PRISMA guidelines and a PICOST framework, a systematic review of white (PubMed, Embase, Cochrane) and gray literature was conducted. Non‑pharmacological interventions examined in the United States (US) and outside the US (OUS) settings included ancillary services (e.g., physical therapy [PT]), assistive devices (e.g., wheelchairs), surgical procedures (e.g., scoliosis correction), and assisted interventions (e.g., enteral feeding).</p><p><strong>Results: </strong>Across 28 eligible studies, patient ages ranged from 1 to 66 years, and 71.4% focused exclusively on female patients with RTT. Approximately 25.0% of the studies were conducted in the US, while 75.0% were conducted in OUS. Six studies examined ancillary service use, where PT utilization ranged from 24.4% to 100.0% and occupational therapy (OT) ranged from 11.5% to 91.7%. Pediatric patients demonstrated a two- to fourfold higher utilization of PT and OT services compared to adults. Among six studies evaluating assistive devices, wheelchair use was reported in up to 90.0% of patients. Nine studies reported surgical procedures, with 1.2%-50.0% of patients requiring scoliosis surgery. Eighteen studies examined assisted interventions, showing enteral feeding utilization rates from 2.0% to 52.0%.</p><p><strong>Conclusion: </strong>Despite the universal clinical reliance on non-pharmacological interventions in RTT, the evidence base quantifying their real-world utilization remains sparse. The wide variability in reported rates might be due to differences in data sources, geographic settings, and patient populations which underscores the need for standardized, prospective, longitudinal research. Age-related declines in ancillary service use and geographic gaps highlight systemic disparities that warrant further investigation. These findings provide a foundational evidence base to inform health resource planning and future research priorities in RTT.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1469-1490"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148138589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Economic Impact of Cumulative Metabolic Comorbidity Burden in Schizophrenia: A Claims-Based Analysis of Medical Costs in the United States.","authors":"Xue Han, Seth C Hopkins, Zhen Zhang, Shivanshu Awasthi","doi":"10.1007/s40120-026-00973-5","DOIUrl":"10.1007/s40120-026-00973-5","url":null,"abstract":"<p><strong>Introduction: </strong>Metabolic comorbidities, including central adiposity, dyslipidemia, insulin resistance, and hypertension, are common in individuals with schizophrenia and contribute to an increased risk of cardiovascular disease and related mortality. There is limited evidence quantifying the economic burden of metabolic comorbidities in schizophrenia. This study aimed to assess the impact of cumulative metabolic comorbidity burden on medical costs in individuals with schizophrenia using claims data.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted using the STATinMED Real World Data Insights database covering the period 2018-2024 in the United States (US). Adults with schizophrenia were grouped by number of metabolic comorbidities (obesity, hyperlipidemia, hypercholesterolemia, diabetes, and/or hypertension). Propensity score matching was applied to balance selected baseline characteristics in individuals with and without metabolic comorbidities. The primary outcome was all-cause medical costs, during a 12-month follow-up, adjusted for insurance status and inflation.</p><p><strong>Results: </strong>Overall, 122,248 individuals were eligible. After matching, the numbers for metabolic comorbidities and individuals in each group were: 0, n = 40,552; 1, n = 15,840; 2, n = 11,833; 3, n = 8119; 4, n = 4074; and 5, n = 686. Estimated total medical costs increased with the number of metabolic comorbidities; individuals with five metabolic comorbidities incurred mean costs of US$34,441 per person per year, over 4 times higher than the $8396 cost for individuals with none. A similar pattern was observed for estimated outpatient, inpatient, and stay-related costs, with 3.5-, 2.3- and 1.7-fold increases, respectively.</p><p><strong>Conclusion: </strong>There is a substantial economic burden associated with cumulative metabolic comorbidities in individuals with schizophrenia. Prevention and management of metabolic comorbidities in this population includes early risk assessments, lifestyle modification, and the selection of antipsychotic medications with few metabolic adverse effects. Addressing metabolic comorbidities should be a key component of multidisciplinary care to reduce health-related and economic impacts in schizophrenia.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2011-2026"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396296/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148259130","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Stability in Cognition and Employment in People with Relapsing Multiple Sclerosis Treated with Cladribine Tablets: Two-year Phase IV CLARIFY-MS Study.","authors":"Bruno Brochet, Dawn Langdon, Eva Kubala Havrdova, Jeanette Lechner-Scott, Xavier Montalban, Francesco Patti, Fredrik Piehl, Alessandra Solari, Raymond Hupperts, Nektaria Alexandri, Annette Lehn, Andrzej Smyk, Krzysztof Selmaj","doi":"10.1007/s40120-026-00897-0","DOIUrl":"10.1007/s40120-026-00897-0","url":null,"abstract":"<p><strong>Introduction: </strong>Cognitive impairment can affect people with multiple sclerosis (MS) at all stages, negatively impacting their work performance and quality of life. This study aimed to report the cognitive function and employment status data for participants with highly active relapsing MS (RMS) treated with cladribine tablets (CladT) during the 2-year, prospective, open-label, exploratory, single-arm, multicentre, phase IV CLARIFY-MS study.</p><p><strong>Methods: </strong>In this post hoc analysis, changes in cognitive function at month (M)12 and M24 (vs baseline) were measured using the Brief International Cognitive Assessment for MS (BICAMS) battery. Additional analyses were conducted to assess clinically meaningful 4- and 8-point changes in Symbol Digit Modalities Test (SDMT) scores. The employment status of participants was determined through a survey at baseline and M24.</p><p><strong>Results: </strong>BICAMS parameter scores remained stable over 2 years in CladT-treated participants. As determined by a 4- and 8-point change, a high proportion of participants had increased or stable SDMT scores at M24 versus baseline. The mean annualised percentage brain volume change (PBVC) in participants was low. No correlation was found between changes in BICAMS parameter scores and annualised PBVC. Furthermore, no major differences in the employment status of participants were observed over 2 years, with > 40% of participants being in full-time employment during the study.</p><p><strong>Conclusions: </strong>Most CLARIFY-MS participants had increased or stable information processing speed at M24 (vs baseline), as determined using clinically meaningful 4- and 8-point changes in SDMT scores. Overall, the cognitive function and employment status of CladT-treated participants with highly active RMS remained stable over 2 years.</p><p><strong>Trial registry details: </strong>URL: https://clinicaltrials.gov/ct2/show/NCT03369665 ; ClinicalTrials.gov Identifier: NCT03369665.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1491-1507"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396085/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434340","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Safety and Effectiveness of 24-Hour Foslevodopa/Foscarbidopa in Parkinson's Disease: ROSSINI Study 6-Month Interim Results.","authors":"Wolfgang H Jost, Filip Bergquist, Andrew Evans, Sharon Hassin-Baer, Robert A Hauser, Tove Henriksen, Irene A Malaty, Tiago A Mestre, Pablo Mir, Ramon Rodriguez, Petra Schwingenschuh, Mihaela Simu, Lars Bergmann, Teresa T Zhou, Sarah Caughlin, Mallika Gopalkrishnan, Pavnit Kukreja, Marie O'Meara, Juan Carlos Parra, Megha B Shah, Jason Aldred","doi":"10.1007/s40120-026-00948-6","DOIUrl":"10.1007/s40120-026-00948-6","url":null,"abstract":"<p><strong>Introduction: </strong>Foslevodopa/foscarbidopa (LDp/CDp) is a nonsurgical 24-h continuous subcutaneous infusion for patients with advanced Parkinson's disease (aPD) and motor fluctuations uncontrolled on oral medications. We present the first multicountry real-world data from routine clinical practice.</p><p><strong>Methods: </strong>ROSSINI (NCT06107426) is an ongoing 3-year multicountry, prospective, observational study of adults with aPD who are LDp/CDp-naïve (cohort A) or transitioning from LDp/CDp open-label extension studies (NCT04379050/NCT04750226, cohort B). For this interim analysis, the primary endpoint was change from baseline to 6 months in OFF time [Movement Disorder Society Unified Parkinson's Disease Rating Scale Part IV (MDS-UPDRS-IV) modified item 4.3]. Safety was assessed by monitoring adverse events (AEs). Interim results for 105 cohort A patients enrolled ≥ 6 months by March 24, 2025 are presented only; cohort B results were limited (n = 5). Mixed-effects models for repeated measurements (continuous outcomes) were utilized, adjusted for country.</p><p><strong>Results: </strong>Cohort A patients had a mean (SD) age of 68.5 (9.5) years, PD duration of 12.1 (5.3) years, and least squares mean (SE) OFF time of 5.2 (0.6) h at baseline. Patients on LDp/CDp showed statistically significant reductions (95% CI) in OFF time [(- 2.8 h (- 3.6, - 1.9), P ≤ .001, n = 47/40 at baseline/month 6], dyskinesia time [- 1.8 h (- 2.6, - 0.9), P ≤ 0.001], MDS-UPDRS-III [- 5.0 (- 8.2, - 1.9), P = 0.002], Parkinson's Disease Sleep Scale-2 [- 5.2 (- 8.0, - 2.4), P ≤ .001], and 39-item Parkinson's Disease Questionnaire [PDQ-39, - 5.6 (- 9.2, - 2.0), P = .002] from baseline to month 6. Freezing of Gait Questionnaire, Gastrointestinal Dysfunction Scale in PD, and King's PD Pain Scale likewise showed statistically significant decreases (P < .05). Overall, 58 (55.2%) reported ≥ 1 AE, primarily nonserious and mild-to-moderate (12.4% serious, 17.1% severe), with hallucinations and infusion site events the most frequently reported events (5.7% each).</p><p><strong>Conclusions: </strong>ROSSINI demonstrates reductions in motor fluctuations and nonmotor symptoms, and increased quality of life in patients with aPD after 6 months of LDp/CDp treatment. The safety profile was consistent with clinical trials.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier, NCT06107426.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1743-1762"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396305/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147840708","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cenobamate: An Appraisal Five Years On.","authors":"Eugen Trinka, John Paul Leach, Josemir W Sander","doi":"10.1007/s40120-026-00944-w","DOIUrl":"10.1007/s40120-026-00944-w","url":null,"abstract":"<p><p>Cenobamate is an antiseizure medication approved in 2021 as add-on therapy for adults with uncontrolled focal seizures. Early trials showed unusually high seizure freedom rates and a favourable tolerability profile, suggesting that cenobamate may represent an important therapeutic advance in the management of focal and other epilepsies. Here, we highlight the latest data from post hoc analyses and clinical studies suggesting that cenobamate is effective across all focal seizure types. It maintains efficacy and safety over the long term, allowing people to reduce their concomitant medications without compromising seizure control. While head-to-head studies are not available, these data, alongside indirect comparisons with other antiseizure medications, support cenobamate as a potentially important advance in the treatment of focal epilepsy.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1451-1467"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396090/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148055860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Steroid-Sparing Effect of Efgartigimod in Generalized Myasthenia Gravis: Study Protocol for a Single-Arm, Open-Label Clinical Trial.","authors":"Tetsu Suzuki, Motoki Fujimaki, Shogo Ohuchi, Takashi Hosaka, Ayako Shioya, Shinji Saiki","doi":"10.1007/s40120-026-00980-6","DOIUrl":"10.1007/s40120-026-00980-6","url":null,"abstract":"<p><strong>Introduction: </strong>Oral corticosteroids remain a cornerstone of therapy for generalized myasthenia gravis (gMG) but are associated with substantial long-term toxicity. Efgartigimod, a human immunoglobulin G1 (IgG1) antibody Fc fragment targeting the neonatal Fc receptor (FcRn), has demonstrated efficacy in gMG; however, no prospective trial has evaluated whether regularly scheduled efgartigimod administration can safely reduce oral prednisolone (PSL) dosage. We designed a prospective clinical study to evaluate the steroid-sparing effect of efgartigimod in patients with gMG who remain dependent on ≥ 6 mg/day of oral PSL.</p><p><strong>Methods: </strong>This is a single-arm, open-label, single-center clinical study. Patients with gMG who are receiving ≥ 6 mg/day of oral PSL on a stable dose for at least 4 weeks prior to enrollment, with any concomitant oral immunosuppressants also at stable doses for at least 4 weeks, and scoring ≥ 5 on the MG Activities of Daily Living (MG-ADL) scale will be enrolled. Efgartigimod will be intravenously administered at 10 mg/kg once weekly for 4 consecutive weeks (one cycle), repeated for four cycles with a 28-day interval between cycles. On the fourth administration day of each cycle, oral PSL will be tapered if MG symptoms have not worsened, defined as no ≥ 1-point increase in the MG-ADL score from both baseline and the first administration day of that cycle.</p><p><strong>Planned outcomes: </strong>The primary endpoint is the change in PSL dosage from baseline at the end of the study. Secondary endpoints include the proportion of patients achieving minimal manifestations (MM) or better status with PSL ≤ 5 mg/day (MM-5 mg), the time to reach ≤ 5 mg/day, the cumulative PSL dose over the study period, the proportion of patients completing the protocol, changes in MG-ADL and other clinical scales, responder rate to efgartigimod, comparison of clinical course between MG subtypes (anti-acetylcholine receptor (AChR) antibody-positive, anti-muscle-specific kinase (MuSK) antibody-positive, and double-seronegative cases) and between patients who are anti-AChR antibody positive, with and without thymoma, changes in indicators of steroid toxicity (HbA1c, lumbar spine bone mineral density, and blood pressure), and the safety profile. The study will also explore biomarkers predictive of efgartigimod response through proteomic and immunologic analyses of serially collected blood samples.  Graphical abstract available for this article.  TRIAL REGISTRATION: Japan Registry of Clinical Trials (jRCT), CRB3180028; registered on 1 October 2024 (prospectively registered).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2167-2180"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396050/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148345953","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multidomain Fatigue, Cognitive, and Quality of Life Observations in Generalized Myasthenia Gravis Under Ravulizumab: A Case Series.","authors":"Aurora Zanghì, Paola Sofia Di Filippo, Claudia Rutigliano, Carlo Avolio, Emanuele D'Amico","doi":"10.1007/s40120-026-00974-4","DOIUrl":"10.1007/s40120-026-00974-4","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to explore fatigue and cognitive features as underrecognized non-motor dimensions in myasthenia gravis (MG), and to describe multidomain observations following initiation of ravulizumab in older adults with generalized myasthenia gravis (gMG).</p><p><strong>Methods: </strong>Three acetylcholine receptor antibody-positive older adults with gMG underwent a standardized multidomain evaluation, including clinical outcomes, patient-reported measures of fatigue and quality of life, and performance-based cognitive assessment. Clinical severity was assessed using the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale and the Quantitative Myasthenia Gravis (QMG) score. Fatigue was assessed using the Neuro-QoL Fatigue scale and the Modified Fatigue Impact Scale (MFIS), and quality of life using the Myasthenia Gravis Quality of Life scale (MG-QoL). Cognitive performance was assessed using the Trail Making Test (TMT). Assessments were performed at baseline and repeated after approximately 12 months and analyzed descriptively.</p><p><strong>Results: </strong>Three patients were enrolled. At baseline, MG-ADL scores ranged from 9 to 10 and QMG scores from 10 to 17. Neuro-QoL Fatigue T-scores ranged from 66 to 70, and MFIS total scores from 18 to 43, with MFIS cognitive subscores ranging from 5 to 21. TMT part B completion times ranged from 284 to 300 s in two patients, while one patient was not evaluable. At follow-up, MG-ADL scores were 7, MG-QoL scores ranged from 10 to 14 (from baseline 22-25), Neuro-QoL Fatigue T-scores were lower by 13-17 points, and MFIS total scores were lower by 3, 20, and 9 points across patients. Changes in MFIS cognitive subscores varied, with one patient showing a slight increase (5 → 6) and others showing reductions. One patient transitioned from non-evaluable to evaluable TMT part B performance, while switching cost showed heterogeneous changes across patients.</p><p><strong>Conclusion: </strong>This exploratory case series describes heterogeneous multidomain trajectories across clinical, fatigue, and cognitive measures in gMG. The findings highlight the feasibility of integrating multidomain assessment of non-motor symptoms in this population, without supporting causal inferences regarding treatment effects.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2181-2188"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396102/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148278259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Observational Retrospective Cohort of Patient Support Program Data on Practical Experiences of Treatment with Foslevodopa/Foscarbidopa in Advanced Parkinson's Disease: The ORCHESTRA Study.","authors":"Tobias Warnecke, Anton Adriaan Van der Plas, Petra Schwingenschuh, Anders Johansson, Mihaela Simu, Marie O'Meara, Resmi Gupta, Bjoern Fritz, Lars Bergmann, Juan Carlos Parra, Martin Südmeyer","doi":"10.1007/s40120-026-00978-0","DOIUrl":"10.1007/s40120-026-00978-0","url":null,"abstract":"<p><strong>Introduction: </strong>Foslevodopa/foscarbidopa (LDp/CDp) is a continuous 24-h subcutaneous infusion therapy for advanced Parkinson's disease (aPD). Clinical trials have demonstrated the efficacy and safety of LDp/CDp; however, real-world evidence is limited.</p><p><strong>Methods: </strong>This retrospective multi-country cohort study used data recorded by AbbVie's patient support program (PSP) nurses during routine interactions with patients with aPD on LDp/CDp (December 2023-February 2025). Patients were followed until earliest of treatment discontinuation or database lock. Patients who started therapy during the study period were included without a pre-defined follow-up duration or standardized scheduled visits.</p><p><strong>Results: </strong>A total of 2590 patients (38.3% female) were followed on average for 141.1 days. During follow-up, mean LDp/CDp base infusion rate was 0.36 (SD = 0.14) mL/h, equivalent to 62.0 (SD = 23.6) mg/h. During the optimization period (mean = 5.4 days), infusion rates generally increased from initial recorded dose to optimization, then remained stable. Among participants with available effectiveness data, 60.0% and 83.0% reported 0 h/day of \"Off\" and dyskinesia symptoms, respectively, in the first month post-optimization; with similar observations over time. 61.1% of patients were assessed as handling the pump well and 80.5% as applying skin care well during the optimization period; both increased post-optimization. The most common reason for discontinuation was infusion site events (5.1%). Exploratory results indicated that younger patients (< 65 years) with shorter disease duration (< 10 years), managed the system better and had more favorable discontinuation outcomes compared to the total population.</p><p><strong>Conclusion: </strong>This study describes real-world use of LDp/CDp in patients with aPD demonstrating that a stable infusion rate was achieved within 5.4 days and provides initial observations on effectiveness. Data completeness varied substantially across variables due to the non-mandatory nature of data collection within the PSP and variable follow up duration. Ongoing efforts to improve data completeness and standardization will support future analyses and enhance understanding of real-world use of LDp/CDp and patient's experience.</p><p><strong>Clinical trial registration: </strong>The study is registered on ClinicalTrials.gov under NCT06937034.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2073-2095"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396073/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148295783","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eculizumab in Myasthenia Gravis: A Multicenter Retrospective Real-World Study in China.","authors":"Yue Li, Huan Yang, Song Tan, Lijun Luo, Xuebing Cao, JinQuan Hu, Bitao Bu, Ting Chang, Zhijun Li","doi":"10.1007/s40120-026-00977-1","DOIUrl":"10.1007/s40120-026-00977-1","url":null,"abstract":"<p><strong>Introduction: </strong>Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disorder needing long-term immunosuppressive therapy. Eculizumab is a promising treatment, but real-world evidence on its efficacy and safety is limited.</p><p><strong>Methods: </strong>This retrospective cohort study across six medical centers enrolled 60 patients with MG on eculizumab. Clinical data, including MG-Activities of Daily Living (MG-ADL) scores, quantitative MG scores (QMG), and oral corticosteroid dosage at baseline, weeks 1, 4, and 8, and final follow-up were obtained. Outcomes included score changes, post-intervention status (PIS), and safety profile.</p><p><strong>Results: </strong>By week 8, 30.95% of patients achieved minimal manifestation status (MMS) or better, increasing to 32.69% at final follow-up. Mean ADL scores significantly dropped from 8.18 ± 3.99 at baseline to 5.59 ± 3.72 at week 1, 3.78 ± 3.42 at week 4, 3.50 ± 2.92 at week 8, and 3.33 ± 3.56 during final follow-up (p < 0.0001). Clinically meaningful improvement (CMI) was observed in 56.9%, 70.0%, 85.0%, and 76.27% at the respective time points. Both thymoma-associated and non-thymoma subgroups showed significant ADL improvement (p ≤ 0.026) with mixed-effects modeling revealing significant group × time interaction (p = 0.01).The thymoma-associated MG (TAMG) group had a faster initial response, with subsequent outcomes comparable between the two groups. Among the 37 patients (61.67%) who switched from FcRn inhibitor therapy to eculizumab, clinical outcomes were non-inferior to those of direct eculizumab recipients and the magnitude of MG-ADL improvement was comparable to that of the overall treatment group. The mean daily corticosteroid dose decreased from 21.21 ± 13.68 mg at baseline to 13.32 ± 10.42 mg at final follow-up (p < 0.0001) Adverse events were rare, with no treatment-related serious events.</p><p><strong>Conclusion: </strong>Eculizumab provides rapid and sustained clinical improvement in patients with MG, with a favorable safety profile. Patients with TAMG show a faster initial response. For patients with an inadequate response to FcRn inhibitors, eculizumab remains an effective rescue strategy.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1973-1992"},"PeriodicalIF":4.7,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13396110/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}