Neurotherapeutics最新文献_第10页

Thermo-Responsive Polysaccharide Nanogels Show Increased Uptake in Niemann-Pick Disease Type C Brain Microvascular Endothelial Cells 热反应性多糖纳米凝胶在尼曼-匹克病C型脑微血管内皮细胞中的摄取增加

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00677

Bilal Moiz , Viviana Alpizar , Eman Mirdamadi , Forbes Porter , Tao Lowe , Alisa Clyne

引用次数: 0

Pridopidine treatment benefits in definite, probable, and early (<18mo onset) subjects with ALS in the HEALEY ALS Platform Trial 在HEALEY ALS平台试验中，Pridopidine治疗对明确、可能和早期（发病<18个月）ALS患者有益处

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00675

Michal Geva , Y Paul Goldberg , Melanie L. Leitner , Kelly Chen , Wei Feng , Noga Gershoni Emek , James D. Berry , Sabrina Paganoni , Jeremy Shefner , Merit ECudkowicz , Michael R. Hayden

引用次数: 0

SLX-100, a novel immediate release formulation of 4-aminopyridine, for the treatment of Spinocerebellar Ataxia 27B (SCA27B), an orphan disease SLX-100是一种新的4-氨基吡啶速释制剂，用于治疗脊髓小脑性共济失调27B (SCA27B)，一种孤儿病

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00687

Amy Chappell M.D.

引用次数: 0

VP3.15, a dual GSK-3β/PDE7 inhibitor, reduces glioblastoma tumor growth though changes in the tumor microenvironment in a PTEN wild-type context VP3.15是一种双重GSK-3β/PDE7抑制剂，在PTEN野生型背景下，通过改变肿瘤微环境来降低胶质母细胞瘤肿瘤的生长。

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00576

Maria Castello-Pons , Maria A. Ramirez-Gonzalez , Patricia Iglesias-Hernández , Nermina Logo Lendo , Carlos Rodriguez-Martín , Laura Quiralte , Juan-Manuel Sepúlveda-Sánchez , Olaya de Dios , Carmen Gil , Ana Martínez , Pilar Sánchez-Gómez , Sergio Casas-Tinto

{"title":"VP3.15, a dual GSK-3β/PDE7 inhibitor, reduces glioblastoma tumor growth though changes in the tumor microenvironment in a PTEN wild-type context","authors":"Maria Castello-Pons , Maria A. Ramirez-Gonzalez , Patricia Iglesias-Hernández , Nermina Logo Lendo , Carlos Rodriguez-Martín , Laura Quiralte , Juan-Manuel Sepúlveda-Sánchez , Olaya de Dios , Carmen Gil , Ana Martínez , Pilar Sánchez-Gómez , Sergio Casas-Tinto","doi":"10.1016/j.neurot.2025.e00576","DOIUrl":"10.1016/j.neurot.2025.e00576","url":null,"abstract":"<div><div>Glioblastoma (GB) is an incurable cancer of the brain, and there is an urgent need to identify effective treatments. This may be achieved by either identifying new molecules or through drug repurposing. To ascertain the therapeutic potential of known GSK-3β and/or PDE7 inhibitors in GB, a drug screening was conducted using a <em>Drosophila melanogaster</em> glioma model. VP3.15, a dual inhibitor with anti-inflammatory and neuroprotective roles in multiple sclerosis, was selected for further investigation. VP3.15 demonstrated robust anti-tumor efficacy against a panel of human and mouse GB cells; however, its capacity to inhibit orthotopic growth was only observed in a wild-type PTEN cell line. The <em>in vivo</em> dependence on PTEN was further suggested with the results in fly gliomas. The analysis of the VP3.15-treated tissues revealed a notable reduction in the number of myeloid cells and in the degree of vascularization. Mechanistic studies indicate that VP3.15 diminishes the production of GAL9, a key molecule that stimulates pro-angiogenic macrophages. Our findings substantiate the pro-tumoral function of GSK-3β, which might depend on the <em>PTEN</em> genetic status. Furthermore, we have delineated the therapeutic potential of VP3.15, which acts through the inhibition of the supportive role of the GB microenvironment. This molecule could be safely and effectively utilized after PTEN characterization in GB patients.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00576"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743461","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}

引用次数: 0

A Real-World Analysis of Stiripentol Discontinuations and Restarts 斯立哌酮停药和重新使用的现实分析

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00662

Kelly R. Gwin PharM.D., Veanna Fong, Fabiola Garcia, Carla Schad

引用次数: 0

Clinical effects of 12-week in-home randomized controlled clinical trial of a novel in-home multifunctional rehabilitation standing and stepping device (MRSSD) vs. static standing desk vs. usual care control groups use in Parkinson's disease (PD) 一种新型家用多功能康复站立行走装置（MRSSD）与静态站立桌、常规护理对照组在帕金森病（PD）治疗中12周的临床随机对照试验

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00669

Nicolaas Ida Bohnen M.D., Ph.D. , Chaetkow Pongmala Ph.D. , Warren VanHout Ph.D. , August Van Hout , Miriam van EM.D.e Boas DP.T.

引用次数: 0

Targeted therapeutic benefits of anakinra for sleep, gastrointestinal, and behavioral symptoms in Sanfilippo syndrome: a post hoc analysis in symptom-specific subgroups 阿那白拉对圣菲利波综合征患者睡眠、胃肠和行为症状的靶向治疗益处：对症状特异性亚组的事后分析

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00679

Jonathan Acevedo BS , Cara O'Neill M.D. , Adolfo Morales M.D. , Michelina Iacovino , Agnes Chen M.D. , Julie Eisengart Ph.D. , Lynda Polgreen M.D., M.S.

引用次数: 0

Advancing Remote Monitoring of Myasthenia Gravis Symptoms through Digital Health Technology 利用数字健康技术推进重症肌无力症状的远程监测

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00683

Ram Kinker Mishra Ph.D. , Ilkay Yildiz Potter Ph.D , Carina Lee Stafstrom Ph.D , Amanda Guidon , Ashkan Vaziri Ph.D.

引用次数: 0

Current perspectives on endovascular therapy for large core ischemic stroke 血管内治疗大面积缺血性脑卒中的最新进展。

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00622

Chloe A. Mutimer, Bruce C.V. Campbell

{"title":"Current perspectives on endovascular therapy for large core ischemic stroke","authors":"Chloe A. Mutimer, Bruce C.V. Campbell","doi":"10.1016/j.neurot.2025.e00622","DOIUrl":"10.1016/j.neurot.2025.e00622","url":null,"abstract":"<div><div>Historically, patients with ischemic stroke and an extensive region of irreversibly injured ischemic core were excluded from endovascular thrombectomy trials due to concerns about limited benefit and high procedural risk. This has fundamentally changed with the publication of five strongly positive randomized controlled trials in this group of patients since 2022 and a sixth trial that showed consistent trends and was positive in per protocol analysis and long-term follow-up. This narrative review summarizes the key findings of these trials, including imaging selection criteria, functional and safety outcomes, and long-term benefits. Across trials, thrombectomy consistently improved functional outcomes at 3-months and 12-months. Absolute functional independence (modified Rankin Scale score 0–2) rates were lower than the trials that enrolled patients with smaller ischemic core volumes, but still significantly favored thrombectomy. Safety outcomes demonstrated a reduction in mortality with EVT and no significant increase in rates of symptomatic intracerebral hemorrhage. While guidelines are being updated to include large core thrombectomy, real-world decision-making remains complex, requiring careful consideration of patient-specific factors, including functional status, infarct location, and patient preferences. Future research should focus on exploring adjunctive therapies and accelerated systems of care to further improve outcomes in this patient population.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00622"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144317527","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}

引用次数: 0

A novel xenon delivery platform provides cerebroprotection during stroke 一种新型氙气输送平台在中风期间提供脑保护

IF 6.9 2区医学

Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00663

Keith Booher

引用次数: 0