Neurotherapeutics最新文献

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Nervonic acid supplementation mitigates disease severity biomarkers in adrenoleukodystrophy 补充神经酸可减轻肾上腺脑白质营养不良患者的疾病严重程度生物标志物。
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-04-07 DOI: 10.1016/j.neurot.2026.e00890
Chenxu Li, Kai Braaten, Ann B. Moser, Wedad Fallatah, Maggie Lorentson, Suzette Huguenin, Anna Torrey, Marcia R. Terluk, Willa Durose, Erin Nolan, Christopher Staley, Subbaya Subramanian, Troy C. Lund, Reena V. Kartha
{"title":"Nervonic acid supplementation mitigates disease severity biomarkers in adrenoleukodystrophy","authors":"Chenxu Li,&nbsp;Kai Braaten,&nbsp;Ann B. Moser,&nbsp;Wedad Fallatah,&nbsp;Maggie Lorentson,&nbsp;Suzette Huguenin,&nbsp;Anna Torrey,&nbsp;Marcia R. Terluk,&nbsp;Willa Durose,&nbsp;Erin Nolan,&nbsp;Christopher Staley,&nbsp;Subbaya Subramanian,&nbsp;Troy C. Lund,&nbsp;Reena V. Kartha","doi":"10.1016/j.neurot.2026.e00890","DOIUrl":"10.1016/j.neurot.2026.e00890","url":null,"abstract":"<div><div>X-linked adrenoleukodystrophy (ALD) is a severe neurometabolic disorder caused by mutations in the <em>ABCD1</em> gene, leading to impaired peroxisomal β-oxidation of very long-chain fatty acids (VLCFAs). The accumulation of saturated VLCFAs, predominantly C26:0, in plasma and across all tissues, contributes to adrenal dysfunction and progressive neurodegeneration. No approved therapy addresses the diverse spectrum of ALD manifestations, underscoring the urgent need for safe, accessible, and preventive treatments. Nervonic acid (NA), a monounsaturated fatty acid, is potentially beneficial for ALD through its neuroprotective effects. Here, we report the safety and therapeutic efficacy of NA in a 4-week dietary intervention study using a mouse model of ALD. NA treatment significantly decreased plasma C26:0-lysophosphatidylcholine, a diagnostic and disease-severity biomarker of ALD, by about 60% as early as one week after intervention. After 4-week treatment, NA markedly reduced free C26:0 and total saturated VLCFA levels in plasma and tissues. Moreover, we observed approximately 56% reduction in brain C26:0-lysophosphatidylcholine levels in NA-fed mice, an effect not reported with other drug intervention. Through comparative microbiome analysis, we show for the first time distinct baseline differences between ALD and wild-type mice, with dietary fatty acid supplementation preventing further dysbiosis. No adverse effects on body weight or food intake were observed throughout the study. Overall, this is the first report demonstrating that an oral dietary fatty acid can ameliorate the hallmark biochemical abnormalities of ALD in plasma and brain, highlighting its potential as a safe and effective therapy, particularly for presymptomatic individuals carrying this genetic defect.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00890"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147639473","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}
引用次数: 0
Current status of the academic invention for neurotherapeutics in Japan 日本神经治疗学学术发明的现状
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e01000
Osamu Kano MD, PhD, Takehisa Hirayama MD, PhD, Mari Shibukawa MD, Naoki Atsuta MD, PhD, Yuishin Izumi MD, PhD, Masashi Aoki MD, PhD
{"title":"Current status of the academic invention for neurotherapeutics in Japan","authors":"Osamu Kano MD, PhD,&nbsp;Takehisa Hirayama MD, PhD,&nbsp;Mari Shibukawa MD,&nbsp;Naoki Atsuta MD, PhD,&nbsp;Yuishin Izumi MD, PhD,&nbsp;Masashi Aoki MD, PhD","doi":"10.1016/j.neurot.2026.e01000","DOIUrl":"10.1016/j.neurot.2026.e01000","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e01000"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148950590","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}
引用次数: 0
Development of an Enzyme Replacement Therapy to treat CLN1 Batten disease: From Bench to Bedside 治疗CLN1巴顿病的酶替代疗法的发展:从实验室到床边
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e00986
Renuka Raman PhD, Ben Horst, Maryam Zare, Jason Winsor, Jixiang Xia, Kendall Case, Thomas Daly, Craig Bencsics, Devin E. Turner, Zahra Shahrokh, Magdalena Leszczyniecka, Emily De Los Reyes, Sean Ekins
{"title":"Development of an Enzyme Replacement Therapy to treat CLN1 Batten disease: From Bench to Bedside","authors":"Renuka Raman PhD,&nbsp;Ben Horst,&nbsp;Maryam Zare,&nbsp;Jason Winsor,&nbsp;Jixiang Xia,&nbsp;Kendall Case,&nbsp;Thomas Daly,&nbsp;Craig Bencsics,&nbsp;Devin E. Turner,&nbsp;Zahra Shahrokh,&nbsp;Magdalena Leszczyniecka,&nbsp;Emily De Los Reyes,&nbsp;Sean Ekins","doi":"10.1016/j.neurot.2026.e00986","DOIUrl":"10.1016/j.neurot.2026.e00986","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00986"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951307","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}
引用次数: 0
Discovery of a novel series of 1,3-cyclodiamines as antagonists of cold (4oC) activation at human TRPM8 发现一系列新的1,3-环二胺作为人TRPM8冷(4oC)激活的拮抗剂
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e00983
Dilipkumar Uredi PhD, Sean Weiner BS, Velvet Journigan PhD
{"title":"Discovery of a novel series of 1,3-cyclodiamines as antagonists of cold (4oC) activation at human TRPM8","authors":"Dilipkumar Uredi PhD,&nbsp;Sean Weiner BS,&nbsp;Velvet Journigan PhD","doi":"10.1016/j.neurot.2026.e00983","DOIUrl":"10.1016/j.neurot.2026.e00983","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00983"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148958796","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}
引用次数: 0
Focused ultrasound in epilepsy 聚焦超声在癫痫中的应用。
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-08-08 DOI: 10.1016/j.neurot.2026.e01030
Ellen J. Bubrick, Lillian H. Stapelfeld, Parker Kotlarz, Nathan J. McDannold, P. Jason White
{"title":"Focused ultrasound in epilepsy","authors":"Ellen J. Bubrick,&nbsp;Lillian H. Stapelfeld,&nbsp;Parker Kotlarz,&nbsp;Nathan J. McDannold,&nbsp;P. Jason White","doi":"10.1016/j.neurot.2026.e01030","DOIUrl":"10.1016/j.neurot.2026.e01030","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e01030"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148697563","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}
引用次数: 0
Preclinical Evaluation of Drug-Induced Sensory Neuropathy: A Veterinary Toxicologic Pathologist’s Perspective 药物性感觉神经病变的临床前评估:兽医毒理学病理学家的观点
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e00988
Deepa B. Rao BVSc, MS, PhD, DABT, DACVP, FIATP
{"title":"Preclinical Evaluation of Drug-Induced Sensory Neuropathy: A Veterinary Toxicologic Pathologist’s Perspective","authors":"Deepa B. Rao BVSc, MS, PhD, DABT, DACVP, FIATP","doi":"10.1016/j.neurot.2026.e00988","DOIUrl":"10.1016/j.neurot.2026.e00988","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00988"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951309","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}
引用次数: 0
NUZ-001 Restores Neurotrophic Support and Enhances Autophagy in Cellular and Zebrafish Models of Huntington’s Disease NUZ-001在亨廷顿舞蹈病的细胞和斑马鱼模型中恢复神经营养支持并增强自噬
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e00992
Jeffrey Brown PhD
{"title":"NUZ-001 Restores Neurotrophic Support and Enhances Autophagy in Cellular and Zebrafish Models of Huntington’s Disease","authors":"Jeffrey Brown PhD","doi":"10.1016/j.neurot.2026.e00992","DOIUrl":"10.1016/j.neurot.2026.e00992","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00992"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951310","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}
引用次数: 0
Clinical Update: NRTX-1001 GABAergic Interneuron Cell Therapy for Drug-Resistant Focal Epilepsy 临床更新:NRTX-1001 gaba能间神经元细胞治疗耐药局灶性癫痫
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e00985
John Hixson MD, Jerry Shih, Sharona Ben-Haim, Robert Beach, Harish Babu, David Spencer, Kim Burchiel, Rebecca O'Dwyer, Sepehr Sani, Derek Southwell, Matthew Luedke, Gautam Banik, Marina Bershteyn, Alessandro Bulfone, Brianna Feld, Luis Fuentealba, Stephanie Hughes, Ji-Hye Jung, Tia Kowal, Sonja Kriks, Cory Nicholas
{"title":"Clinical Update: NRTX-1001 GABAergic Interneuron Cell Therapy for Drug-Resistant Focal Epilepsy","authors":"John Hixson MD,&nbsp;Jerry Shih,&nbsp;Sharona Ben-Haim,&nbsp;Robert Beach,&nbsp;Harish Babu,&nbsp;David Spencer,&nbsp;Kim Burchiel,&nbsp;Rebecca O'Dwyer,&nbsp;Sepehr Sani,&nbsp;Derek Southwell,&nbsp;Matthew Luedke,&nbsp;Gautam Banik,&nbsp;Marina Bershteyn,&nbsp;Alessandro Bulfone,&nbsp;Brianna Feld,&nbsp;Luis Fuentealba,&nbsp;Stephanie Hughes,&nbsp;Ji-Hye Jung,&nbsp;Tia Kowal,&nbsp;Sonja Kriks,&nbsp;Cory Nicholas","doi":"10.1016/j.neurot.2026.e00985","DOIUrl":"10.1016/j.neurot.2026.e00985","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e00985"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951318","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}
引用次数: 0
Post Hoc Analysis of RESCUE Trial Shows Shortened Hospital Stay and Higher Rate of Discharge to Home in Participants Treated with RNS60 救援试验的事后分析显示,接受RNS60治疗的参与者住院时间缩短,出院率更高
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e01016
Supurna Ghosh, Jordan S. Dubow, Jocelyn Sutherland, Andreas Kalmes, Jarrad Mock, Sameer A. Ansari, Christopher G. Favilla, David Chiu, Wayne M. Clark, David S. Liebeskind, Ryan A. McTaggart
{"title":"Post Hoc Analysis of RESCUE Trial Shows Shortened Hospital Stay and Higher Rate of Discharge to Home in Participants Treated with RNS60","authors":"Supurna Ghosh,&nbsp;Jordan S. Dubow,&nbsp;Jocelyn Sutherland,&nbsp;Andreas Kalmes,&nbsp;Jarrad Mock,&nbsp;Sameer A. Ansari,&nbsp;Christopher G. Favilla,&nbsp;David Chiu,&nbsp;Wayne M. Clark,&nbsp;David S. Liebeskind,&nbsp;Ryan A. McTaggart","doi":"10.1016/j.neurot.2026.e01016","DOIUrl":"10.1016/j.neurot.2026.e01016","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e01016"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951434","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}
引用次数: 0
Human immunocompetent 3D neural spheroid assay platform for high throughput screening and therapeutic development 用于高通量筛选和治疗开发的人类免疫三维神经球体检测平台
IF 7.4 2区 医学
Neurotherapeutics Pub Date : 2026-07-01 Epub Date: 2026-09-09 DOI: 10.1016/j.neurot.2026.e01007
Jiajing Zhang PhD, Yi Wei Lim PhD, Angelica Medina PhD, Yu-Chi Chen, Martin Carrasco Carvajal, Marc Ferrer PhD, Emily Lee PhD
{"title":"Human immunocompetent 3D neural spheroid assay platform for high throughput screening and therapeutic development","authors":"Jiajing Zhang PhD,&nbsp;Yi Wei Lim PhD,&nbsp;Angelica Medina PhD,&nbsp;Yu-Chi Chen,&nbsp;Martin Carrasco Carvajal,&nbsp;Marc Ferrer PhD,&nbsp;Emily Lee PhD","doi":"10.1016/j.neurot.2026.e01007","DOIUrl":"10.1016/j.neurot.2026.e01007","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"23 4","pages":"Article e01007"},"PeriodicalIF":7.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148951515","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}
引用次数: 0
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