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J: Clinical therapeutics最新文献

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J06 Pivot-HD: a phase 2, randomised, placebo-controlled study to evaluate the safety and efficacy of ptc518 in subjects with Huntington’s disease J06 Pivot-HD:一项2期、随机、安慰剂对照研究，评估ptc518在亨廷顿病患者中的安全性和有效性
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.256
B. Beers, A. Bredlau, Anusuya Bhattacharyya, A. Kristensen, L. Golden, M. Klein, S. Peltz
{"title":"J06 Pivot-HD: a phase 2, randomised, placebo-controlled study to evaluate the safety and efficacy of ptc518 in subjects with Huntington’s disease","authors":"B. Beers, A. Bredlau, Anusuya Bhattacharyya, A. Kristensen, L. Golden, M. Klein, S. Peltz","doi":"10.1136/jnnp-2022-ehdn.256","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.256","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"25 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":"129028033","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
J11 Pridopidine exerts beneficial effects in preclinical models and in clinical trials via activation of the sigma-1 receptor, in a biphasic manner J11 Pridopidine通过激活sigma-1受体在临床前模型和临床试验中以双相方式发挥有益作用
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.261
M. Geva, Deniz Akinc Abdulhayoglu, Shao-Ming Wang, Noga Gershoni-Emek, B. Boulanger, Y. P. Goldberg, T. Su, M. R. Hayden
{"title":"J11 Pridopidine exerts beneficial effects in preclinical models and in clinical trials via activation of the sigma-1 receptor, in a biphasic manner","authors":"M. Geva, Deniz Akinc Abdulhayoglu, Shao-Ming Wang, Noga Gershoni-Emek, B. Boulanger, Y. P. Goldberg, T. Su, M. R. Hayden","doi":"10.1136/jnnp-2022-ehdn.261","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.261","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"12 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":"131650291","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 Quantitative analyses of pooled tominersen clinical data to support dose selection (rationale) for the new phase II study J02对汇总的临床数据进行定量分析，以支持新的II期研究的剂量选择(基本原理)
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.252
Yumi Yamamoto, Julian Zhou, P. Grimsey, P. McColgan, P. Ducray
{"title":"J02 Quantitative analyses of pooled tominersen clinical data to support dose selection (rationale) for the new phase II study","authors":"Yumi Yamamoto, Julian Zhou, P. Grimsey, P. McColgan, P. Ducray","doi":"10.1136/jnnp-2022-ehdn.252","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.252","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"25 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":"125739005","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
J03 Evaluating tominersen effect on self-reported quality of life (QoL) with high-frequency longitudinal, digital euroqol 5-dimensions 5-level (EQ-5D-5L) survey in generation HD1, a phase III trial of tominersen in adult individuals with manifest Huntington’s disease (HD) 通过高频纵向、数字euroqol 5维5水平(EQ-5D-5L)调查评估tominersen对HD1代自我报告生活质量(QoL)的影响，这是一项在成年明显亨廷顿病(HD)患者中进行的tominersen III期试验
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.253
D. Berchtold, Johannes Rennig, Cedric Simillion, F. Lipsmeier, P. McColgan, M. Lindemann, Jonas F. Dorn
{"title":"J03 Evaluating tominersen effect on self-reported quality of life (QoL) with high-frequency longitudinal, digital euroqol 5-dimensions 5-level (EQ-5D-5L) survey in generation HD1, a phase III trial of tominersen in adult individuals with manifest Huntington’s disease (HD)","authors":"D. Berchtold, Johannes Rennig, Cedric Simillion, F. Lipsmeier, P. McColgan, M. Lindemann, Jonas F. Dorn","doi":"10.1136/jnnp-2022-ehdn.253","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.253","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"12 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":"131961914","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
J01 The EHDN advanced therapies working group (ATWG) J01 EHDN先进疗法工作组(ATWG)
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.251
R. Aron Badin, W. Gray, M. Busse, A. Perrier, V. Wheelock, S. Goldman, J. M. Canals, L. Thompson, A. Rosser
{"title":"J01 The EHDN advanced therapies working group (ATWG)","authors":"R. Aron Badin, W. Gray, M. Busse, A. Perrier, V. Wheelock, S. Goldman, J. M. Canals, L. Thompson, A. Rosser","doi":"10.1136/jnnp-2022-ehdn.251","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.251","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"36 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":"115727717","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
J18 Guided self-help for anxiety among people with Huntington’s disease compared to treatment as usual: a randomised controlled feasibility trial (GUIDE-HD) 与常规治疗相比，亨廷顿舞蹈症患者焦虑的引导自助:一项随机对照可行性试验(GUIDE-HD)
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.268
M. Dale, F. Eccles, Katie Melvin, Zaynah Khan, L. Jones, N. Zarotti, R. Kiani, J. Simpson
{"title":"J18 Guided self-help for anxiety among people with Huntington’s disease compared to treatment as usual: a randomised controlled feasibility trial (GUIDE-HD)","authors":"M. Dale, F. Eccles, Katie Melvin, Zaynah Khan, L. Jones, N. Zarotti, R. Kiani, J. Simpson","doi":"10.1136/jnnp-2022-ehdn.268","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.268","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"45 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":"114974389","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
J07 Synthesis of key results and conclusions of the signal phase 2 study of pepinemab as a treatment for early HD J07 pepinemab治疗早期HD信号2期研究关键结果及结论综合
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.257
M. Zauderer, E. Evans, T. Fisher, Vikas Mishra, J. Leonard, D. Oakes, Kimberly Walters, Lisa Kowarski, E. Siemers, K. Kieburtz, A. Feigin
{"title":"J07 Synthesis of key results and conclusions of the signal phase 2 study of pepinemab as a treatment for early HD","authors":"M. Zauderer, E. Evans, T. Fisher, Vikas Mishra, J. Leonard, D. Oakes, Kimberly Walters, Lisa Kowarski, E. Siemers, K. Kieburtz, A. Feigin","doi":"10.1136/jnnp-2022-ehdn.257","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.257","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"5 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":"121825073","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
J12 The perspective program: evaluating the effect of SAGE-718 on cognitive function in patients with Huntington’s disease 展望项目:评估SAGE-718对亨廷顿舞蹈病患者认知功能的影响
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.262
A. Koenig, Julie Stout, J. Johannesen, J. Petrillo, Sigui Li, Pranoti Pradhan, Emily Lefler, K. Paumier, J. Wald, M. Quirk, J. Doherty
{"title":"J12 The perspective program: evaluating the effect of SAGE-718 on cognitive function in patients with Huntington’s disease","authors":"A. Koenig, Julie Stout, J. Johannesen, J. Petrillo, Sigui Li, Pranoti Pradhan, Emily Lefler, K. Paumier, J. Wald, M. Quirk, J. Doherty","doi":"10.1136/jnnp-2022-ehdn.262","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.262","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"27 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":"125733264","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
J10 Analysis of integrated safety data from pridopidine clinical trials demonstrates a favorable safety and tolerability profile at the clinically relevant dose of 45 mg bid J10对普利多啶临床试验综合安全性数据的分析表明，在临床相关剂量为45mg / bid时，其安全性和耐受性良好
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.260
M. Geva, B. Darpo, G. Ferber, Y. Paul Goldberg, R. Reilmann, M. R. Hayden
{"title":"J10 Analysis of integrated safety data from pridopidine clinical trials demonstrates a favorable safety and tolerability profile at the clinically relevant dose of 45 mg bid","authors":"M. Geva, B. Darpo, G. Ferber, Y. Paul Goldberg, R. Reilmann, M. R. Hayden","doi":"10.1136/jnnp-2022-ehdn.260","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.260","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"35 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":"122713293","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
J16 Exploring the acceptability and key components for success of a 12 month physical activity intervention in early to mid-stage Huntington’s disease J16探讨早期至中期亨廷顿舞蹈病12个月体育活动干预成功的可接受性和关键因素
J: Clinical therapeutics Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.266
C. Drew, K. Hamana, R. Williams-Thomas, R. Playle, M. Busse, L. Quinn
{"title":"J16 Exploring the acceptability and key components for success of a 12 month physical activity intervention in early to mid-stage Huntington’s disease","authors":"C. Drew, K. Hamana, R. Williams-Thomas, R. Playle, M. Busse, L. Quinn","doi":"10.1136/jnnp-2022-ehdn.266","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.266","url":null,"abstract":"","PeriodicalId":251913,"journal":{"name":"J: Clinical therapeutics","volume":"304 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":"127564779","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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