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J: Clinical therapeutics最新文献

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J13 Psychotropic medication use in Huntinton’s disease: a retrospective cohort study 精神药物在亨廷顿病中的应用:一项回顾性队列研究
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.263
R. L. Andriessen, M. Oosterloo, A. Hollands, D. E. Linden, B. T. A. Greef, A. Leentjens
{"title":"J13 Psychotropic medication use in Huntinton’s disease: a retrospective cohort study","authors":"R. L. Andriessen, M. Oosterloo, A. Hollands, D. E. Linden, B. T. A. Greef, A. Leentjens","doi":"10.1136/jnnp-2022-ehdn.263","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.263","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"18 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"121617705","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J04 Benchmarking enroll-HD to a phase III placebo cohort from generation HD1 to inform clinical trial planning 从HD1代开始，将入组hd患者纳入安慰剂III期队列，为临床试验计划提供信息
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.254
H. Furby, Athanasios Siadimas, P. McColgan, L. Boak, L. Polito, E. Neacy, J. Long, Cristina Sampaio
{"title":"J04 Benchmarking enroll-HD to a phase III placebo cohort from generation HD1 to inform clinical trial planning","authors":"H. Furby, Athanasios Siadimas, P. McColgan, L. Boak, L. Polito, E. Neacy, J. Long, Cristina Sampaio","doi":"10.1136/jnnp-2022-ehdn.254","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.254","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122322863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J17 HD-DRUM – a novel computerised drumming training for movement and cognitive abilities in people with Huntington’s disease – app development and protocol of a randomised controlled feasibility study J17 HD-DRUM -一种针对亨廷顿舞蹈病患者运动和认知能力的新型计算机击鼓训练-应用程序开发和随机对照可行性研究的协议
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.267
C. Metzler-Baddeley, M. Busse, C. Drew, P. Pallmann, Derek K Jones, A. Rosser
{"title":"J17 HD-DRUM – a novel computerised drumming training for movement and cognitive abilities in people with Huntington’s disease – app development and protocol of a randomised controlled feasibility study","authors":"C. Metzler-Baddeley, M. Busse, C. Drew, P. Pallmann, Derek K Jones, A. Rosser","doi":"10.1136/jnnp-2022-ehdn.267","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.267","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"84 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"115777477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J08 A phase 2 open label study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ANX005 in patients with, or at risk of, manifest Huntington’s disease (HD) J08一项2期开放标签研究，评估明显亨廷顿舞蹈病(HD)患者或有风险患者静脉注射ANX005的安全性、耐受性、药代动力学和药效学
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.258
Rajeev Kumar, D. Claassen, A. Mongan, Benjamin Hoehn, P. Lin, E. Cahir-McFarland, L. Taylor, Priya Chandra, T. Yednock, H. Kroon
{"title":"J08 A phase 2 open label study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ANX005 in patients with, or at risk of, manifest Huntington’s disease (HD)","authors":"Rajeev Kumar, D. Claassen, A. Mongan, Benjamin Hoehn, P. Lin, E. Cahir-McFarland, L. Taylor, Priya Chandra, T. Yednock, H. Kroon","doi":"10.1136/jnnp-2022-ehdn.258","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.258","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"15 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"116861816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J09 An update on the proof-HD phase 3 trial: pridopidine’s outcome on function in huntington [09]亨廷顿舞蹈症3期临床试验的最新进展:哌啶对亨廷顿舞蹈症功能的影响
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.259
R. Reilmann, A. Feigin, A. Rosser, S. Kostyk, M. Geva, Noga Gershoni-Emek, D. Boot, Yael Cohen, Y. P. Goldberg, M. R. Hayden
{"title":"J09 An update on the proof-HD phase 3 trial: pridopidine’s outcome on function in huntington","authors":"R. Reilmann, A. Feigin, A. Rosser, S. Kostyk, M. Geva, Noga Gershoni-Emek, D. Boot, Yael Cohen, Y. P. Goldberg, M. R. Hayden","doi":"10.1136/jnnp-2022-ehdn.259","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.259","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"362 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132931674","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J15 Akathisia as a side effect of tetrabenazine treatment in HD patients [15]丁苯那嗪治疗HD患者的副作用:静坐障碍
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.265
Karolina Ziora-Jakutowicz, I. Stępniak, Grzegorz Witkowski, A. Sułek, Ewelina Elert‐Dobkowska, W. Krysa
{"title":"J15 Akathisia as a side effect of tetrabenazine treatment in HD patients","authors":"Karolina Ziora-Jakutowicz, I. Stępniak, Grzegorz Witkowski, A. Sułek, Ewelina Elert‐Dobkowska, W. Krysa","doi":"10.1136/jnnp-2022-ehdn.265","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.265","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132372520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J05 Interim results from cohort 1 of the double-blind, dose-escalation phase I/II clinical trial of amt-130 (HD-genetrx-1) for early-stage huntington’s disease (HD) amt-130 (HD-genetrx-1)治疗早期亨廷顿舞蹈病(HD)的双盲、剂量递增I/II期临床试验队列1的中期结果
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.255
E. Furr Stimming, V. Sung, C. Testa, S. Kostyk, C. Ross, A. Samii, M. Geschwind, Deborah Hall, P. Dayalu, R. Lonser, Brad Elder, P. Larson, David L. Cooper, Marcie Clarkin, T. Ali, R. Dolmetsch
{"title":"J05 Interim results from cohort 1 of the double-blind, dose-escalation phase I/II clinical trial of amt-130 (HD-genetrx-1) for early-stage huntington’s disease (HD)","authors":"E. Furr Stimming, V. Sung, C. Testa, S. Kostyk, C. Ross, A. Samii, M. Geschwind, Deborah Hall, P. Dayalu, R. Lonser, Brad Elder, P. Larson, David L. Cooper, Marcie Clarkin, T. Ali, R. Dolmetsch","doi":"10.1136/jnnp-2022-ehdn.255","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.255","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"129949221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
J14 Subcutaneous haloperidol for chorea in patients with Huntington’s disease and dysphagia 皮下氟哌啶醇治疗亨廷顿舞蹈病和吞咽困难患者的舞蹈病
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.264
Sarah Lee, Indrajith Jayasinghe, Y. Lie
{"title":"J14 Subcutaneous haloperidol for chorea in patients with Huntington’s disease and dysphagia","authors":"Sarah Lee, Indrajith Jayasinghe, Y. Lie","doi":"10.1136/jnnp-2022-ehdn.264","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.264","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"83 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"123097069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J04 Efficacy and safety of tiapride for chorea treatment in huntington’s disease: a systematic review [04]替阿必利治疗舞蹈病的疗效和安全性:系统评价
J: Clinical therapeutics Pub Date : 2021-09-01 DOI: 10.1136/jnnp-2021-ehdn.131
S. Feleus, Malu van Schaijk, R. Roos, Susanne T de Bot
{"title":"J04 Efficacy and safety of tiapride for chorea treatment in huntington’s disease: a systematic review","authors":"S. Feleus, Malu van Schaijk, R. Roos, Susanne T de Bot","doi":"10.1136/jnnp-2021-ehdn.131","DOIUrl":"https://doi.org/10.1136/jnnp-2021-ehdn.131","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"11 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"131606857","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
J02 Trident: investigating the safety and feasibility of fetal cell transplants in huntingtons disease 胎儿细胞移植治疗亨廷顿病的安全性和可行性研究
J: Clinical therapeutics Pub Date : 2021-09-01 DOI: 10.1136/jnnp-2021-ehdn.129
C. Drew, F. Sharouf, E. Randell, L. Brookes-Howell, Kim Smallman, B. Sewell, A. Burrell, N. Kirby, Laura Mills, S. Precious, P. Pallmann, D. Gillespie, K. Hood, M. Busse, W. Gray, A. Rosser
{"title":"J02 Trident: investigating the safety and feasibility of fetal cell transplants in huntingtons disease","authors":"C. Drew, F. Sharouf, E. Randell, L. Brookes-Howell, Kim Smallman, B. Sewell, A. Burrell, N. Kirby, Laura Mills, S. Precious, P. Pallmann, D. Gillespie, K. Hood, M. Busse, W. Gray, A. Rosser","doi":"10.1136/jnnp-2021-ehdn.129","DOIUrl":"https://doi.org/10.1136/jnnp-2021-ehdn.129","url":null,"abstract":"Background The relatively focal and specific loss of striatal medium spiny neurons in Huntington’s disease (HD) makes the disorder a suitable candidate for cell replacement therapy (CRT), a process involving the transplantation of donor cells to replace those lost due to disease. Whilst previous trials of CRT in HD have shown proof of principle, further investigation to demonstrate ongoing safety and efficacy is warranted. Aim Assess the safety and feasibility of transplanting an increased number of human fetal striatal cells into the striatum of people with HD. Methods TRIDENT (TRIal DEsigns for delivery of Novel Therapies in neurodegeneration) is an open label phase 1 trial using a Trial Within a Cohort (TWiC) design. A minimum of 18 participants will be enrolled in the study observational cohort, and up to five eligible participants will be randomly selected to undergo transplantation of 12-22 million fetal cells in a dose escalation paradigm. Independent reviewers will assess safety outcomes (lack of significant infection, bleeding or new neurological deficit) four weeks after surgery, and ongoing safety will be established before conducting each subsequent surgery. All participants will undergo detailed clinical and functional assessment at baseline, 6 and 12 months. Surgery will be performed one month after baseline, and transplant participants will undergo regular clinical follow-up for at least 12 months. Results 20 participants have been recruited to the observational cohort. The first transplant surgery is scheduled for July 2021. Conclusion The data collected in TRIDENT will; 1) enable assessment of the safety and feasibility of fetal cell transplants; 2) inform trial designs for complex intracranial interventions in a range of neurodegenerative conditions and 3) facilitate the development of stable surgical pipelines for delivery of future stem cell trials.","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"72 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"116173838","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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